CIBMTR Scientific July 1, Working Committee 2019 ...• If Working Committee Leadership clears the proposal to move forward, the MS-level Statistician contacts the study investigator

CIBMTR Scientific Working Committee

Research Portfolio

July 1, 2019

Milwaukee Campus Medical College of Wisconsin

9200 W Wisconsin Ave, Suite C5500 Milwaukee, WI 53226 USA

(414) 805-0700

Minneapolis Campus National Marrow Donor Program/

Be The Match – 500 N 5th St Minneapolis, MN 55401-9959 USA

(763) 406-5800

cibmtr.org

CIBMTR Scientific Working Committee Research Portfolio: July 1, 2019

TABLE OF CONTENTS 1.0 OVERVIEW .................................................................................................................................................................. 1

1.1 Membership ........................................................................................................................................................... 2 1.2 Leadership .............................................................................................................................................................. 2 1.3 Productivity ............................................................................................................................................................ 3 1.4 How to Get Involved ............................................................................................................................................ 3

2.0 ACUTE LEUKEMIA WORKING COMMITTEE .................................................................................................. 6 2.1 Leadership .............................................................................................................................................................. 6 2.2 Recent Publications ............................................................................................................................................. 6 2.3 Current Studies ..................................................................................................................................................... 9

3.0 CELLULAR IMMUNOTHERAPY FOR CANCER WORKING COMMITTEE ............................................13 3.1 Leadership ............................................................................................................................................................13 3.2 Recent Publications ...........................................................................................................................................13 3.3 Current Studies ...................................................................................................................................................14

4.0 CHRONIC LEUKEMIA WORKING COMMITTEE ...........................................................................................16 4.1 Leadership ............................................................................................................................................................16 4.2 Recent Publications ...........................................................................................................................................16 4.3 Current Studies ...................................................................................................................................................19

5.0 DONOR HEALTH AND SAFETY WORKING COMMITTEE ........................................................................23 5.1 Leadership ............................................................................................................................................................23 5.2 Recent Publications ...........................................................................................................................................23 5.3 Current Studies ...................................................................................................................................................26

6.0 GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE ......................................................29 6.1 Leadership ............................................................................................................................................................29 6.2 Recent Publications ...........................................................................................................................................29 6.3 Current Studies ...................................................................................................................................................32

7.0 GRAFT-VS-HOST DISEASE WORKING COMMITTEE .................................................................................35 7.1 Leadership ............................................................................................................................................................35 7.2 Recent Publications ...........................................................................................................................................35 7.3 Current Studies ...................................................................................................................................................40

8.0 HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE .............................42 8.1 Leadership ............................................................................................................................................................42 8.2 Recent Publications ...........................................................................................................................................42 8.3 Current Studies ...................................................................................................................................................45

9.0 IMMUNOBIOLOGY WORKING COMMITTEE ................................................................................................49 9.1 Leadership ............................................................................................................................................................49 9.2 Recent Publications ...........................................................................................................................................49 9.3 Current Studies ...................................................................................................................................................56

2019 CIBMTR Scientific Working Committee Research Portfolio TABLE OF CONTENTS

10.0 INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE .......................................66 10.1 Leadership ..........................................................................................................................................................66 10.2 Recent Publications ........................................................................................................................................66 10.3 Current Studies .................................................................................................................................................68

11.0 LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE .....................................................70 11.1 Leadership ..........................................................................................................................................................70 11.2 Recent Publications ........................................................................................................................................70 11.3 Current Studies .................................................................................................................................................76

12.0 LYMPHOMA WORKING COMMITTEE ..........................................................................................................79 12.1 Leadership ..........................................................................................................................................................79 12.2 Recent Publications ........................................................................................................................................79 12.3 Current Studies .................................................................................................................................................84

13.0 NON-MALIGNANT DISEASES WORKING COMMITTEE .........................................................................87 13.1 Leadership ..........................................................................................................................................................87 13.2 Recent Publications ........................................................................................................................................87 13.3 Current Studies .................................................................................................................................................90

14.0 PEDIATRIC CANCER WORKING COMMITTEE ..........................................................................................93 14.1 Leadership ..........................................................................................................................................................93 14.2 Recent Publications ........................................................................................................................................93 14.3 Current Studies .................................................................................................................................................94

15.0 PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE ..................96 15.1 Leadership ..........................................................................................................................................................96 15.2 Recent Publications ........................................................................................................................................96 15.3 Current Studies .............................................................................................................................................. 100

16.0 REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE ............... 104 16.1 Leadership ....................................................................................................................................................... 104 16.2 Recent Publications ..................................................................................................................................... 104 16.3 Current Studies .............................................................................................................................................. 108

APPENDIX A: COLLABORATIVE STUDIES ......................................................................................................... 111 APPENDIX B: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ................................................. 117 APPENDIX C: GUIDELINES FOR STUDY PRINCIPAL INVESTIGATORS .................................................. 120 APPENDIX D: GLOSSARY ......................................................................................................................................... 125

2019 CIBMTR Scientific Working Committee Research Portfolio 1.0 OVERVIEW

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1.0 OVERVIEW The CIBMTR® (Center for International Blood and Marrow Transplant Research®) collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy research worldwide to increase survival and enrich quality of life for patients. A research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin, the CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of participating centers, a unique and extensive clinical database, and a robust and comprehensive biospecimen repository.

Fifteen international Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research. Each committee focuses on a specific disease or condition, use of cellular therapy, or complication of treatment (Table 1). This report details the Scientific Working Committee Research Portfolio as of July 1, 2019.

Table 1. Working Committee Focus Areas

Working Committee Scientific Focus

Acute Leukemia HCT for acute leukemia and pre-leukemia Cellular Immunotherapy for Cancer

Non-transplant uses of hematopoietic stem cells

Chronic Leukemia HCT for chronic leukemias, myelodysplastic disorders, and myeloproliferative disorders

Donor Health and Safety Donor safety and outcomes Graft Sources and Manipulation Graft types, composition, and manipulation techniques Graft-versus-Host Disease Biology, prevention, and treatment of graft-versus-host

disease and its complications Health Services and International Studies

Social and economic barriers to HCT access, including quality of care and the influence of psychosocial factors on transplant outcomes, as well as international issues and differences in HCT

Immunobiology Histocompatibility and other genetic and immunologic issues related to HCT

Infection and Immune Reconstitution

Prevention and treatment of post-transplant infections and issues related to recovery of immune function

Late Effects and Quality of Life

Long-term survival after HCT, including clinical and psychosocial effects of transplantation

Lymphoma HCT for Hodgkin and non-Hodgkin lymphoma Non-Malignant Diseases HCT and cellular therapy for non-malignant diseases,

including autoimmune diseases, inherited and acquired marrow failure, hemoglobinopathy, immunodeficiency diseases, and inborn errors of metabolism


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Working Committee Scientific Focus

Pediatric Cancer HCT for childhood leukemias and other issues related to use of HCT in children

Plasma Cell Disorders and Adult Solid Tumors

HCT for multiple myeloma and other plasma cell disorders as well as solid tumors in adults

Regimen-Related Toxicity and Supportive Care

Preparative regimens, prevention, and treatment of early non-graft-versus-host disease toxicities; supportive care in the early post-transplant period

1.1 Membership

Total Working Committee membership exceeds 2,800 researchers. Membership is open to any researcher willing to take an active role in developing and conducting studies that use CIBMTR data and / or resources. While most of these individuals are HCT clinicians, statisticians and basic scientists also participate. PhD Statistical Directors and Master’s-level statisticians from the CIBMTR Coordinating Center provide their unique expertise in data analysis. Basic scientists investigating HLA, immunogenetics, pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective research areas. The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities.

1.2 Leadership

Each Working Committee is staffed by at least one MS-level Statistician, a PhD Statistical Director, and an MD Scientific Director from the CIBMTR Coordinating Center. Each also typically has two to four Chairs who are appointed by the Advisory Committee.

1.2.1 Committee Chairs

Working Committee Chairs are appointed by the Advisory Committee to non-renewable five-year terms. Appointments are made each fall, with terms commencing on March 1 of the following year. Terms are staggered to facilitate succession and maintain continuity. Individuals may serve as Chair more than once but not consecutively for the same committee. Chairs participate in the nomination process for replacement positions and give special consideration to promising junior investigators, thus promoting ongoing leadership for the work of the CIBMTR.

Working Committee Chairs provide subject matter expertise in cellular therapy as well as understanding of CIBMTR organization and procedures. They must be members of CIBMTR centers that submit comprehensive report form (CRF)-level data and are compliant with continuous process improvement standards for data submission, unless an exception is granted by the Advisory Committee. Chairs are occasionally selected from outside these guidelines for their specific scientific expertise, for example, a scientist who directs a histocompatibility laboratory, apheresis center, or donor registry, who is committed to the CIBMTR and to the field of cellular therapy.


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Chairs monitor and facilitate the progress of studies in their Working Committee’s portfolio. They communicate with principal investigators (PIs) to address barriers and / or delays and participate in weekly CIBMTR Coordinating Center study critiques when studies in their portfolios are being discussed. In addition to chairing annual Working Committee meetings, Chairs meet by teleconference every four to six weeks with their committee’s Scientific Director and biostatisticians to review the progress of study proposals and ongoing studies. Chairs lead the annual Working Committee meeting, and, using input from that meeting, they prioritize studies and establish the research agenda for the following year.

1.3 Productivity

There are currently 181 studies in progress, 63 of which are collaborations with other organizations (Appendix A). At the 2019 TCT Meetings, 39 new study proposals were approved. The prioritization and selection process (Figure 1) ensures the most important issues can be addressed in a timely manner.

During the past year, Working Committee study investigators published 54 manuscripts in peer-reviewed journals, approximately half of the total number of CIBMTR publications. In each committee’s section of this report, publications since July 1, 2014, are listed (Sections 2.2-16.2). For a complete list of CIBMTR publications, visit the CIBMTR Publication List webpage.

Working Committee study investigators presented 33 abstracts (16 oral and 17 poster) at national and international conferences this year. These presentations include 22 (13 oral and 9 poster) at the 2018 American Society of Hematology Annual Meeting, 9 (2 oral and 7 poster) at the 2019 TCT Meetings, and 2 (1 oral and 1 poster) at other conferences.

1.4 How to Get Involved

Working Committees are collaborative in nature, and all interested individuals are encouraged to participate:

• Join a Working Committee. Learn more about each committee on the CIBMTR Working Committee webpage. To join a Working Committee, email [email protected], contact the Working Committee leadership listed on the individual committee’s webpage, or attend a Working Committee Meeting at the TCT | Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and CIBMTR (TCT Meetings).

• Attend a Working Committee Meeting at the TCT Meetings. All TCT Meeting attendees may attend to learn more about the committee, its recent publications and current studies, and have the opportunity to learn about and provide feedback on new study proposals.

• Participate in a Writing Committee. When a draft protocol is approved by the Working Committee leadership and Coordinating Center, all Working Committee members on record are invited to participate in the study Writing Committee.

• Propose a Study. Anyone willing to follow the study development and management process (Appendix B) is eligible to propose a study to the Working Committees (Figure

https://www.cibmtr.org/ReferenceCenter/PubList/Pages/index.aspx

https://www.cibmtr.org/About/WhoWeAre/Committees/

https://www.cibmtr.org/About/WhoWeAre/Committees/

mailto:[email protected]


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1). Guidelines for CIBMTR study PIs, including hints and tips to make the study process as successful as possible, are provided in Appendix C.

For more information regarding participation in a Working Committee, access the “Learn more about how to get involved in a Working Committee” section on the CIBMTR Working Committee webpage.

https://www.cibmtr.org/About/WhoWeAre/Committees/wc/Pages/default.aspx

https://www.cibmtr.org/About/WhoWeAre/Committees/wc/Pages/default.aspx


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Figure 1. Working Committee Study Proposal Review Process

Submission

• By mid-November, study investigator submits proposal to the CIBMTR Coordinating Center for consideration at the next TCT Meetings.

InitialReview

• Working Committee Leadership reviews for feasibility with CIBMTR data, potential conflict with active studies, scientific merit, and ability to complete the study in a timely fashion. Researchers with similar concepts may be advised to combine their proposals.

Preliminary Assessment

• If Working Committee Leadership clears the proposal to move forward, the MS-level Statistician contacts the study investigator and prepares a table of characteristics of patient data based on the population defined in the proposal.

Presentation

• Study investigator presents the proposal at the Working Committee meeting at the February TCT Meetings.

Voting

• Working Committee members vote for each proposal, assigning a scientific impact score to each.

Final Approval

• Working Committee Leadership utilizes member feedback in determining which proposals to pursue. Advisory Committee approves the CIBMTR research agenda.

Notification

• Working Committee Leadership contacts study investigator to notify of study approval / rejection by the beginning of April.

2019 CIBMTR Scientific Working Committee Research Portfolio 2.0 ACUTE LEUKEMIA WORKING COMMITTEE

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2.0 ACUTE LEUKEMIA WORKING COMMITTEE

2.1 Leadership

Chair: Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Email: [email protected] Chair: Mark Litzow, MD, Mayo Clinic Rochester Email: [email protected] Chair: Partow Kebriaei, MD, MD Anderson Cancer Center Email: [email protected] Scientific Director: Daniel Weisdorf, MD, CIBMTR Minneapolis Email: [email protected] Asst Sci Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Mei-Jie Zhang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Karen Chen , MS, CIBMTR Milwaukee Email: [email protected]

2.2 Recent Publications

2019

Rashidi A, Hamadani M, Zhang M-J, Wang H-L, Abdel-Azim H, Aljurf M, Assal A, Bajel A, Bashey A, Battiwalla M, Beitinjaneh AM, Bejanyan N, Bhatt VR, Bolaños-Meade J, Byrne M, Cahn J-Y, Cairo M, Ciurea S, Copelan E, Cutler C, Daly A, Diaz M-A, Farhadfar N, Gale RP, Ganguly S, Grunwald MR, Hahn T, Hashmi S, Hildebrandt GC, Holland HK, Hossain N, Kanakry CG, Kharfan-Dabaja MA, Khera N, Koc Y, Lazarus HM, Lee JW, Maertens J, Martino R, McGuirk J, Munker R, Murthy HS, Nakamura R, Nathan S, Nishihori T, Palmisiano N, Patel S, Pidala J, Olin R, Olsson RF, Oran B, Ringden O, Rizzieri D, Rowe J, Savoie ML, Schultz KR, Seo S, Schaffer BC, Singh A, Solh M, Stockerl-Goldstein K, Verdonck LF, Wagner J, Waller EK, de Lima M, Sandmaier BM, Litzow MR, Weisdorf D, Romee R, Saber W. Outcomes of haploidentical vs matched sibling transplantation for acute myeloid leukemia in first complete remission. Blood Advances. 2019 Jun 25; 3(12):1826-1836. doi:10.1182/bloodadvances.2019000050. Epub 2019 Jun 14. ncbi.nlm.nih.gov/pubmed/31201170

Ustun C, Le-Rademacher J, Wang H-L, Othus M, Sun Z, Major B, Zhang M-J, Storrick E, Lafky JM, Chow S, MrÃ³zek K, Attar EC, Nand S, Bloomfield CD, Cripe LD, Tallman MS, Appelbaum F, Larson RA, Marcucci G, Roboz GJ, Uy GL, Stone RM, Jatoi A, Shea TC, de Lima M, Foran JM, Sandmaier BM, Litzow MR, Erba HP, Hurria A, Weisdorf DJ, Artz A. Allogeneic hematopoietic cell transplantation compared to chemotherapy consolidation in older acute myeloid leukemia (AML) patients 60-75 years in first complete remission (CR1): An alliance (A151509), SWOG, ECOG-ACRIN, and CIBMTR study. Leukemia. doi:10.1038/s41375-019-0477-x. Epub 2019 May 9. ncbi.nlm.nih.gov/pubmed/31073153








http://www.ncbi.nlm.nih.gov/pubmed/31201170



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Yeshurun M, Weisdorf D, Rowe JM, Tallman MS, Zhang M-J, Wang HL, Saber W, de Lima M, Sandmaier BM, Uy G, Kamble RT, Cairo MS, Cooper BW, Cahn J-Y, Ganguly S, Camitta B, Verdonck LF, Dandoy C, Diaz MA, Savani BN, George B, Liesveld J, McGuirk J, Byrne M, Grunwald MR, Drobyski WR, Pulsipher MA, Abdel-Azim H, Prestidge T, Wieduwilt MJ, Martino R, Norkin M, Beitinjaneh A, Seo S, Nishihori T, Wirk B, Frangoul H, Bashey A, Mori S, Marks DI, Bachanova V. The impact of the graft-versus-leukemia effect on survival in acute lymphoblastic leukemia. Blood Advances. 2019 Feb 26; 3(4):670-680. doi:10.1182/bloodadvances. 2018027003. Epub 2019 Feb 26. PMC6391668. ncbi.nlm.nih.gov/pubmed/30808685

2018

Eapen M, Brazauskas R, Hemmer M, Perez WS, Steinert P, Horowitz MM, Deeg HJ. Hematopoietic cell transplant for acute myeloid leukemia and myelodysplastic syndrome: Conditioning regimen intensity. Blood Advances. 2018 Aug 28; 2(16):2095-2103. doi:10.1182/ bloodadvances.2018021980. Epub 2018 Aug 22. PMC6113615. ncbi.nlm.nih.gov/pubmed/ 30135184

2017

Bejanyan N, Zhang M-J, Wang H-L, Lazaryan A, de Lima M, Marks DI, Sandmaier BM, Bachanova V, Rowe J, Tallman M, Kebriaei P, Kharfan-Dabaja M, Gale RP, Lazarus HM, Ustun C, Copelan E, Hamilton BK, Schiller G, Hogan W, Hashmi S, Seftel M, Kanakry CG, Olsson RF, Martino R, Saber W, Khoury HJ, Weisdorf DJ. Pretransplant consolidation is not beneficial for adults with ALL undergoing myeloablative allogeneic transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 May 1; 24(5):945-955. doi:10.1016/j.bbmt.2017.12.784. Epub 2017 Dec 21. PMC5953798. ncbi.nlm.nih.gov/pubmed/29275139

Kebriaei P, Anasetti C, Zhang M-J, Wang H-L, Aldoss I, de Lima M, Khoury HJ, Sandmaier BM, Horowitz MM, Artz A, Bejanyan N, Ciurea S, Lazarus HM, Gale RP, Litzow M, Bredeson C, Seftel MD, Pulsipher MA, Boelens J-J, Alvarnas J, Champlin R, Forman S, Pullarkat V, Weisdorf D, Marks DI. Intravenous busulfan compared with total body irradiation pretransplant conditioning for adults with acute lymphoblastic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Apr 1; 24(4):726-733. doi:10.1016/j.bbmt.2017.11.025. Epub 2017 Dec 2. PMC5902420. ncbi.nlm.nih.gov/ pubmed/29197676

Ustun C, Giannotti F, Zhang M-J, Wang H-L, Brunstein C, Labopin M, Rocha V, de Lima M, Baron F, Sandmaier B, Eapen M, Gluckman E, Nagler A, Weisdorf DJ, Ruggeri A. Outcomes of UCB transplantation are comparable in FLT3+ AML: Results of CIBMTR, EUROCORD and EBMT collaborative analysis. Leukemia. 2017 Jun 1; 31(6):1408-1414. doi:10.1038/leu.2017.42. Epub 2017 Jan 25. PMC5462854. ncbi.nlm.nih.gov/pubmed/28119528

Segal E, Martens M, Wang HL, Brazauskas R, Weisdorf D, Sandmaier BM, Khoury HJ, de Lima M, Saber W. Comparing outcomes of matched related donor and matched unrelated donor hematopoietic cell transplants in adults with B-Cell acute lymphoblastic leukemia. Cancer. 2017 Sep 1; 123(17):3346-3355. doi:10.1002/cncr.30737. Epub 2017 Apr 27. PMC5568918. ncbi.nlm.nih.gov/pubmed/28452054










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Weisdorf DJ, Millard HR, Horowitz MM, Hyare PS, Champlin R, Ho V, Mielcarek M, Rezvani A, Stockerl-Goldstein K, Khoury HJ, De Lima M, Saber W, Sandmaier B, Zhang M-J, Eapen M. Allogeneic transplantation for advanced acute myeloid leukemia: The value of complete remission. Cancer. 2017 Jun 1; 123(11):2025-2034. doi:10.1002/cncr.30536. Epub 2017 Jan 24. PMC5445001. ncbi.nlm.nih.gov/pubmed/28117884

Michelis FV, Gupta V, Zhang M-J, Wang H-L, Aljurf M, Bacher U, Beitinjaneh A, Chen Y-B, DeFilipp Z, Gale RP, Kebriaei P, Kharfan-Dabaja M, Lazarus HM, Nishihori T, Olsson RF, Oran B, Rashidi A, Rizzieri DA, Tallman MS, de Lima M, Khoury HJ, Sandmaier BM, Weisdorf D, Saber W. Cytogenetic risk determines outcomes after allogeneic transplantation in older patients with acute myeloid leukemia in their second complete remission: A Center for International Blood and Marrow Transplant Research cohort analysis. Cancer. 2017 Jun 1; 123(11):2035-2042. doi:10.1002/cncr.30567. Epub 2017 Jan 24. PMC5445018. ncbi.nlm.nih.gov/pubmed/28117898

2016

Rosko AE, Wang H-L, de Lima M, Sandmaier B, Khoury HJ, Artz A, Brammer J, Bredeson C, Farag S, Kharfan-Dabaja M, Lazarus HM, Marks DI, Martino Bufarull R, McGuirk J, Mohty M, Nishihori T, Nivison-Smith I, Rashidi A, Ringden O, Seftel M, Weisdorf D, Bachanova V, Saber W. Reduced intensity conditioned allograft yields favorable survival for older adults with B-cell acute lymphoblastic leukemia. American Journal of Hematology. 2017 Jan 1; 92(1):42-49. doi:10.1002/ajh.24575. Epub 2016 Nov 12. PMC5167625. ncbi.nlm.nih.gov/pubmed/27712033

Deol A, Sengsayadeth S, Ahn KW, Wang H-L, Aljurf M, Antin JH, Battiwalla M, Bornhauser M, Cahn J-Y, Camitta B, Chen Y-B, Cutler CS, Gale RP, Ganguly S, Hamadani M, Inamoto Y, Jagasia M, Kamble R, Koreth J, Lazarus HM, Liesveld J, Litzow MR, Marks DI, Nishihori T, Olsson RF, Reshef R, Rowe JM, Saad AA, Sabloff M, Schouten HC, Shea TC, Soiffer RJ, Uy GL, Waller EK, Wiernik PH, Wirk B, Woolfrey AE, Bunjes D, Devine S, de Lima M, Sandmaier BM, Weisdorf D, Khoury HJ, Saber W. Does FLT3 mutation impact survival after hematopoietic stem cell transplantation for acute myeloid leukemia? A Center for International Blood and Marrow Transplant Research (CIBMTR) analysis. Cancer. doi:10.1002/cncr.30140. Epub 2016 Jun 17. PMC5030151. ncbi.nlm.nih.gov/pubmed/27315441

Ganzel C, Mathews V, Alimoghaddam K, Ghavamzadeh A, Kuk D, Devlin S, Wang H, Zhang M-J, Weisdorf D, Douer D, Rowe JM, Polge E, Esteve J, Nagler A, Mohty M, Tallman M. Autologous transplant remains the preferred therapy for relapsed APL in CR2. Bone Marrow Transplantation. doi:10.1038/bmt.2016.96. Epub 2016 Apr 18. PMC5014591. ncbi.nlm.nih.gov/pubmed/27088379

Seftel MD, Neuberg D, Zhang M-J, Wang H-L, Ballen KK, Bergeron J, Couban S, Freytes CO, Hamadani M, Kharfan-Dabaja MA, Lazarus HM, Nishihori T, Paulson K, Saber W, Sallan SE, Soiffer R, Tallman MS, Woolfrey AE, DeAngelo DJ, Weisdorf DJ. Pediatric-inspired therapy compared to allografting for Philadelphia chromosome-negative adult ALL in first complete remission. American Journal of Hematology. 2016 Mar 1; 91(3):322-329. doi:10.1002/ ajh.24285. Epub 2016 Feb 22. PMC4764423. ncbi.nlm.nih.gov/pubmed/26701142



http://www.ncbi.nlm.nih.gov/





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Munker R, Brazauskas R, Wang HL, de Lima M, Khoury HJ, Gale RP, Maziarz RT, Sandmaier BM, Weisdorf D, Saber W. Allogeneic hematopoietic cell transplantation for patients with mixed phenotype acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jun 1; 22(6):1024-1029. doi:10.1016/j.bbmt.2016.02.013. Epub 2016 Feb 21. PMC4867266. ncbi.nlm.nih.gov/pubmed/ 26903380

2015

Pasquini MC, Zhang M-J, Medeiros BC, Armand P, Hu Z-H, Nishihori T, Aljurf MD, Akpek G, Cahn J-Y, Cairo MS, Cerny J, Copelan EA, Deol A, Freytes CO, Gale RP, Ganguly S, George B, Gupta V, Hale GA, Kamble RT, Klumpp TR, Lazarus HM, Luger SM, Liesveld JL, Litzow MR, Marks DI, Martino R, Norkin M, Olsson RF, Oran B, Pawarode A, Pulsipher MA, Ramanathan M, Reshef R, Saad AA, Saber W, Savani BN, Schouten HC, Ringdén O, Tallman MS, Uy GL, Wood WA Jr, Wirk B, Pérez WS, Batiwalla M, Weisdorf DJ. Hematopoietic cell transplantation outcomes in monosomal karyotype myeloid malignancies. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Feb 1; 22(2):248-257. doi:10.1016/j.bbmt.2015.08.024. Epub 2015 Aug 29. PMC4716890. ncbi.nlm.nih.gov/pubmed/ 26327629

Goyal SD, Zhang MJ, Wang HL, Akpek G, Copelan EA, Freytes C, Gale RP, Hamadani M, Inamoto Y, Kamble RT, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Reshef R, Ritchie DS, Saber W, Savani BN, Seber A, Shea TC, Tallman MS, Wirk B, Bunjes DW, Devine SM, de Lima M, Weisdorf DJ, Uy GL. Allogeneic hematopoietic cell transplant for AML: No impact of pre-transplant extramedullary disease on outcome. Bone Marrow Transplantation. 2015 Aug 1; 50(8):1057-1062. doi:10.1038/bmt.2015.82. Epub 2015 Apr 27. PMC4527880. ncbi.nlm.nih.gov/pubmed/ 25915806

2014

Bejanyan N, Weisdorf DJ, Logan BR, Wang H-L, Devine SM, de Lima M, Bunjes DW, Zhang M-J. Survival of patients with acute myeloid leukemia relapsing after allogeneic hematopoietic cell transplantation: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Mar 1; 21(3):454-459. doi:10.1016/j.bbmt.2014.11.007. Epub 2014 Nov 15. PMC4329076. ncbi.nlm.nih.gov/pubmed/25460355

2.3 Current Studies

LK13-02 Title: Prognostic significance of cytogenetic abnormalities in patients with

Philadelphia-negative acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation in complete remission

PIs: Aleksandr Lazaryan (H. Lee Moffitt Cancer Center and Research Institute) Veronika Bachanova (University of Minnesota Blood and Marrow Transplant Program) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020)









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LK15-03 Title: Comparison of outcomes of older adolescents and young adults with Philadelphia-

chromosome/BCR-ABL1-negative acute lymphoblastic leukemia receiving post-remission consolidation chemotherapy with pediatric-inspired chemotherapy on CALGB 10403 or myeloablative allogeneic hematopoietic cell transplantation

PIs: Matthew Wieduwilt (University of California, San Diego Medical Center) Wendy Stock (University of Chicago Medicine) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with CALGB 10403 LK16-01 Title: Reduced intensity conditioning regimens for acute myeloid leukemia: A

comparison of busulfan and melphalan based regimens from the CIBMTR database

PIs: Zartash Gul (University of Cincinnati Medical Center) Hassan Alkhateeb (Mayo Clinic Rochester) Rajneesh Nath (Banner MD Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LK16-02 Title: DRI-guided choice of conditioning intensity for allogeneic hematopoietic cell

transplantation in adults with acute myeloid leukemia and myelodysplastic syndromes

PIs: Nelli Behanyan (H. Lee Moffitt Cancer Center and Research Institute) Erica Warlick (University of Minnesota Blood and Marrow Transplant Program) Claudio Brunstein (University of Minnesota Blood and Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LK16-03 Title: Allogeneic transplantation to treat therapy related acute myeloid leukemia and

myelodysplastic syndromes PIs: Natalie Callander (University of Wisconsin Hospital and Clinics) Leland Metheny (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Marcos De Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Aric Hall (University of Wisconsin Hospital and Clinics) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020)


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LK17-01 Title: Outcomes of acute myeloid leukemia patients who undergo allogeneic transplant

stratified by depth of clinical response PIs: Mary-Elizabeth Percival (University of Washington) Brenda Sandmaier (Fred Hutchinson Cancer Research Center) Eli Estey (M.D. Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LK17-02 Title: Allogeneic hematopoietic transplant outcomes in adult patients with MLL-

rearranged acute myeloid leukemia PIs: Kamal Menghrajani (Memorial Sloan Kettering Cancer Center) Martin Tallman (Memorial Sloan Kettering Cancer Center) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) LK17-03 Title: Impact of post-transplant maintenance therapy with BCR-ABL tyrosine kinase

inhibitors on outcomes of Philadelphia chromosome-positive acute lymphoblastic leukemia

PIs: Zack DeFilipp (Massachusetts General Hospital) Yi-Bin Chen (Massachusetts General Hospital) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) LK18-01 Title: Prognostic impact of the new European LeukemiaNet genetic risk stratification

categories in predicting outcomes for adults with acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation

PIs: Antonio Jimenez (University of Miami) Trent Wang (Fox Chase Temple University Hospital Bone Marrow Transplant Program) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Krishna Komanduri (University of Miami) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020)


Page | 12

LK18-02 Title: Comparison of outcomes of HCT with matched-related donor or matched-

unrelated donor alloHCT for adults with acute lymphoblastic leukemia PIs: Matthew Wieduwilt (University of California, San Diego Medical Center) Leland Metheny (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) LK19-01 Title: Evaluating outcomes of hematopoietic cell transplantation in blastic plasmacytoid

dendritic cell neoplasm PI: Hemant Murthy (Mayo Clinic Florida) Status: Protocol Pending (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) LK19-02 Title: Evolving significance of Ph-chromosome status on ALL prognosis in the TKI era PIs: Maxwell Krem (James Graham Brown Cancer Center, University of Louisville School of

Medicine) Richard Maziarz (Oregon Health and Science University) Status: Protocol Pending (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020)

LK19-03 Title: Outcomes of alloHCT in AML patients who achieved first complete remission after

two or more cycles of induction chemotherapty PIs: Michael Boyiadzis (University of Pittsburgh Medical Center - Cancer Center) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Status: Protocol Pending (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 3.0 CELLULAR IMMUNOTHERAPY FOR CANCER WORKING COMMITTEE

Page | 13

3.0 CELLULAR IMMUNOTHERAPY FOR CANCER WORKING COMMITTEE

3.1 Leadership

Chair: Sarah Nikiforow, MD, PhD, Dana Farber Cancer Institute Email: [email protected] Chair: Peiman Hematti, MD, University of Wisconsin Hospital and Clinics Email: [email protected] Scientific Director: Marcelo Pasquini, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Xianmiao Qiu , MS, CIBMTR Milwaukee Email: [email protected]


2018

Sullivan KM, Majhail NS, Bredeson C, Carpenter PA, Chatterjee S, Crofford LJ, Georges GE, Nash RA, Pasquini MC, Sarantopoulos S, Storek J, Savani B, St. Clair EW. Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: Position statement from the American Society for Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.06.025. Epub 2018 Jun 25. ncbi.nlm.nih.gov/pubmed/29953945

2017

Muraro PA, Pasquini M, Atkins HL, Bowen JD, Farge D, Fassas A, Freedman MS, Georges GE, Gualandi F, Hamerschlak N, Havrdova E, Kimiskidis VK, Kozak T, Mancardi GL, Massacesi L, Moraes DA, Nash RA, Pavletic S, Ouyang J, Rovira M, Saiz A, Simoes B, Trnný M, Zhu L, Badoglio M, Zhong X, Sormani MP, Saccardi R. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurology. 2017 Apr 1; 74(4):459-469. doi:10.1001/jamaneurol.2016.5867. Epub 2017 Feb 20. PMC5744858. ncbi.nlm.nih.gov/ pubmed/28241268










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3.3 Current Studies

AC16-01 Title: Pattern of use and outcomes with donor lymphocyte infusion after HLA-

haploidentical allogeneic hematopoietic stem cell transplant PIs: Eva Gupta (Mayo Clinic Florida) James Foran (Mayo Clinic Florida) Vivek Roy (Mayo Clinic Florida) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) AC17-01 Title: CD-19 chimeric antigen receptor T cells with or without hematopoietic cell

transplantation for treatment of refractory acute lymphocytic leukemia PIs: Miguel-Angel Perales (Memorial Sloan Kettering Cancer Center) Jae Park (Memorial Sloan Kettering Cancer Center) Sarah Nikiforow (Dana Farber Cancer Institute) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) AC18-01 Title: Effect of stem cell boost and donor lymphocyte infusion on the incidence of GVHD PIs: James Yoon (Emory University Hospital) Edmund Waller (Emory University) Status: Draft Protocol Received (as of July 1, 2019)

Protocol Development (expected by June 30, 2020) CT13-01 Title: Utility of donor leukocyte infusion for the treatment of drug-resistant viral or

fungal infections in allogeneic HCT recipients: A CIBMTR analysis PI: Gorgun Akpek (Rush University Medical Center) Status: Analysis (as of July 1, 2019)


CT19-01 Title: Allogeneic hematopoietic cell transplantation vs chimeric antigen receptor t-cell

therapy for DLBCL patients with prior autologous transplant failure or refractory disease

PIs: Mehdi Hamadani (Medical College of Wisconsin) Marcelo Pasquini (Medical College of Wisconsin) Frederick Locke (H. Lee Moffitt Cancer Center and Research Institute) Ajay Gopal (Fred Hutchinson Cancer Research Center) Status: Protocol Pending (as of July 1, 2019)



Page | 15

CT19-02 Title: Prolonged cytopenia following CD-19 targeted CAR-T therapy for diffuse large B-

cell lymphoma PI: Mazyar Shadman (Fred Hutchinson Cancer Research Center) Status: Protocol Pending (as of July 1, 2019)

Submitted (expected by June 30, 2020) CT19-03 Title: Patient outcomes after CAR-T cells PI: Marcelo Pasquini (Medical College of Wisconsin) Status: Protocol Development (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

Page | 16

4.0 CHRONIC LEUKEMIA WORKING COMMITTEE

4.1 Leadership

Chair: Ronald Sobecks, MD, Cleveland Clinic Foundation Email: [email protected] Chair: Bart Scott, MD, Fred Hutchinson Cancer Research Center Email: [email protected] Chair: Ryotaro Nakamura, MD, City of Hope Email: [email protected] Scientific Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Directors: Kwang Woo Ahn, PhD, CIBMTR Milwaukee Email: [email protected] Soyoung Kim, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Noel Estrada-Merly, MPH, CIBMTR Milwaukee Email: [email protected]


2019

Kim HT, Ahn KW, Hu Z-H, Davids MS, Volpe VO, Antin JH, Sorror ML, Shadman M, Press O, Pidala J, Hogan W, Negrin R, Devine S, Uberti J, Agura E, Nash R, Mehta J, McGuirk J, Forman S, Langston A, Giralt SA, Perales M-A, Battiwalla M, Hale GA, Gale RP, Marks DI, Hamadani M, Ganguly S, Bacher U, Lazarus H, Reshef R, Hildebrandt GC, Inamoto Y, Cahn J-Y, Solh M, Kharfan-Dabaja MA, Ghosh N, Saad A, Aljurf M, Schouten HC, Hill BT, Pawarode A, Kindwall-Keller T, Saba N, Copelan EA, Nathan S, Beitinjaneh A, Savani BN, Cerny J, Grunwald MR, Yared J, Wirk BM, Nishihori T, Chhabra S, Olsson RF, Bashey A, Gergis U, Popat U, Sobecks R, Alyea E, Saber W, Brown JR. Prognostic score and cytogenetic risk classification for chronic lymphocytic leukemia patients: Center for International Blood and Marrow Transplant Research report. Clinical Cancer Research. 2019 Aug 15; 26(16):5143-5155. doi:10.1158/1078-0432.CCR-18-3988. Epub 2019 Jun 28. PMC6697588. ncbi.nlm.nih.gov/pubmed/31253630

2018

Chhabra S, Ahn KW, Hu Z-H, Jain S, Assal A, Cerny J, Copelan EA, Daly A, DeFilipp Z, Gadalla SM, Gale RP, Ganguly S, Hamilton BK, Hildebrandt GC, Hsu JW, Inamoto Y, Kanate AS, Khoury HJ, Lazarus HM, Litzow MR, Nathan S, Olsson RF, Pawarode A, Ringden O, Rowe JM, Saad A, Savani BN, Schouten HC, Seo S, Shah NN, Solh M, Stuart RK, Ustun C, Woolfrey AE, Yared JA, Alyea EP, Kalaycio ME, Popat U, Sobecks R, Saber W. Myeloablative vs reduced-intensity conditioning allogeneic hematopoietic cell transplantation for chronic myeloid leukemia. Blood Advances. 2018 Nov 13; 2(21):2922-2936. doi:10.1182/bloodadvances.2018024844. Epub 2018 Nov 5. PMC6234373. ncbi.nlm.nih.gov/pubmed/30396912








https://www.ncbi.nlm.nih.gov/pubmed/31253630



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2017

Hill BT, Ahn KW, Hu Z-H, Aljurf M, Beitinjaneh A, Cahn J-Y, Cerny J, Kharfan-Dabaja MA, Ganguly S, Ghosh N, Grunwald MR, Inamoto Y, Kindwall-Keller T, Nishihori T, Olsson RF, Saad A, Seftel M, Seo S, Szer J, Tallman M, Ustun C, Wiernik PH, Maziarz RT, Kalaycio M, Alyea E, Popat U, Sobecks R, Saber W. Assessment of impact of HLA type on outcomes of allogeneic hematopoietic stem cell transplantation for chronic lymphocytic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Mar 1; 24(3):581-586. doi:10.1016/j.bbmt.2017.10.015. Epub 2017 Oct 12. PMC5944847. ncbi.nlm.nih.gov/pubmed/29032274

Gerds AT, Woo Ahn K, Hu ZH, Abdel-Azim H, Akpek G, Aljurf M, Ballen KK, Beitinjaneh A, Bacher U, Cahn J-Y, Chharbra S, Cutler C, Daly A, DeFilipp Z, Gale RP, Gergis U, Grunwald MR, Hale GA, Hamilton BK, Jagasia M, Kamble RT, Kindwell-Keller T, Nishihori T, Olsson RF, Ramanathan M, Saad AA, Solh M, Ustun C, Valcárcel D, Warlick E, Wirk BM, Kalaycio M, Alyea E, Popat U, Sobecks R, Saber W. Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jun 1; 23(6):971-979. doi:10.1016/j.bbmt. 2017.03.014. Epub 2017 Mar 10. PMC5474679. ncbi.nlm.nih.gov/pubmed/28288952

Liu HD, Ahn KW, Hu Z-H, Hamadani M, Nishihori T, Wirk B, Beitinjaneh A, Rizzieri D, Grunwald MR, Sabloff M, Olsson RF, Bajel A, Bredeson C, Daly A, Inamoto Y, Majhail N, Saad A, Gupta V, Gerds A, Malone A, Tallman M, Reshef R, Aljurf M, Marks DI, Copelan E, Gergis U, Savoie ML, Ustun C, Litzow MR, Cahn JY, Kindwell-Keller T, Akpek G, Savani BN, Rowe JM, Wiernik PH, Hsu JW, Cortes J, Kalaycio M, Maziarz R, Sobecks R, Popat U, Alyea E, Saber W. Allogeneic hematopoietic cell transplant for adult chronic myelomonocytic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2017.01.078. Epub 2017 Jan 20. ncbi.nlm.nih.gov/ pubmed/28115276

2016

Saber W, Horowitz MM. Transplantation for myelodysplastic syndromes: Who, when, and which conditioning regimens. Hematology / the Education Program of the American Society of Hematology. 2016 Dec 2; 2016(1):478-484. doi:10.1182/asheducation-2016.1.478. ncbi.nlm.nih.gov/pubmed/27913519

Kharfan-Dabaja MA, Kumar A, Hamadani M, Stilgenbauer S, Ghia P, Anasetti C, Dreger P, Montserrat E, Perales M-A, Alyea EP, Awan FT, Ayala E, Barrientos JC, Brown JR, Castro JE, Furman RR, Gribben J, Hill BT, Mohty M, Moreno C, O'Brien S, Pavletic SZ, Pinilla-Ibarz J, Reddy NM, Sorror M, Bredeson C, Carpenter P, Savani BN. Clinical practice recommendations for use of allogeneic hematopoietic cell transplantation in chronic lymphocytic leukemia on behalf of the Guidelines Committee of the American Society for Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Dec 1; 22(12):2117-2125. doi:10.1016/ j.bbmt.2016.09.013. Epub 2016 Sep 19. PMC5116249. ncbi.nlm.nih.gov/pubmed/27660167








Page | 18

Ustun C, Gotlib J, Popat U, Artz A, Litzow M, Reiter A, Nakamura R, Kluin-Nelemans HC, Verstovsek S, Gajewski J, Perales M-A, George T, Shore T, Sperr W, Saber W, Kota V, Yavuz AS, Pullarkat V, Rogosheske J, Hogan W, Van Besien K, Hagglund H, Damaj G, Arock M, Horny H-P, Metcalfe DD, Deeg HJ, Devine S, Weisdorf D, Akin C, Valent P. Consensus opinion on allogeneic hematopoietic cell transplantation in advanced systemic mastocytosis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1348-1356. doi:10.1016/j.bbmt.2016.04.018. Epub 2016 Apr 27. ncbi.nlm.nih.gov/pubmed/27131865

Shaffer BC, Ahn KW, Hu Z-H, Nishihori T, Malone AK, Valcárcel D, Grunwald MR, Bacher U, Hamilton B, Kharfan-Dabaja MA, Saad A, Cutler C, Warlick E, Reshef R, Wirk BM, Sabloff M, Fasan O, Gerds A, Marks D, Olsson R, Wood WA, Costa LJ, Miller AM, Cortes J, Daly A, Kindwall-Keller TL, Kamble RT, Rizzieri DA, Cahn J-Y, Gale RP, William B, Litzow M, Wiernik PH, Liesveld J, Savani BN, Vij R, Ustun C, Copelan E, Popat U, Kalaycio M, Maziarz R, Alyea E, Sobecks R, Pavletic S, Tallman M, Saber W. Scoring system prognostic of outcome in patients undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome. Journal of Clinical Oncology. doi:10.1200/JCO.2015.65.0515. Epub 2016 Apr 4. ncbi.nlm.nih.gov/pubmed/ 27044940

Chaudhury S, Sparapani R, Hu ZH, Nishihori T, Abdel-Azim H, Malone A, Olsson R, Hamadani M, Daly A, Bacher U, Wirk BM, Kamble RT, Gale RP, Wood WA, Hale G, Wiernik PH, Hashmi SK, Marks DI, Ustun C, Munker R, Savani BN, Alyea E, Popat U, Sobecks R, Kalaycio M, Maziarz R, Burlington, Wisconsin 53105Hijiya N, Saber W. Outcomes of allogeneic hematopoietic cell transplantation in children (<18y) and young adults (18-29y) with chronic myeloid leukemia: A CIBMTR cohort analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jun 1; 22(6):1056-1064. doi:10.1016/j.bbmt.2016.02.015. Epub 2016 Mar 8. PMC4877686. ncbi.nlm.nih.gov/ pubmed/26964698

2014

Copelan EA, Avalos BR, Ahn KW, Zhu X, Gale RP, Grunwald MR, Hamadani M, Hamilton BK, Hale GA, Marks DI, Waller EK, Savani BN, Costa LJ, Ramanathan M, Cahn J-Y, Khoury HJ, Weisdorf DJ, Inamoto Y, Kamble RT, Schouten HC, Wirk B, Litzow MR, Aljurf MD, van Besien KW, Ustun C, Bolwell BJ, Bredeson CN, Fasan O, Ghosh N, Horowitz MM, Arora M, Szer J, Loren AW, Alyea EP, Cortes J, Maziarz RT, Kalaycio ME, Saber W. Comparison of outcomes of allogeneic transplantation for chronic myeloid leukemia with cyclophosphamide in combination with intravenous busulfan, oral busulfan, or total body irradiation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Mar 1; 21(3):552-558. doi:10.1016/j.bbmt.2014.12.010. Epub 2014 Dec 17. PMC4329042. ncbi.nlm.nih.gov/pubmed/25528388








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4.3 Current Studies

CK12-01 Title: Optimal timing of allogeneic stem cell transplantation for chronic myeloid

leukemia patients in the tyrosine kinase inhibitor era PI: Hans Lee (M.D. Anderson Cancer Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with M.D. Anderson Cancer Center CK15-01 Title: Comparison of transplant versus non-transplant therapies for myelofibrosis PIs: Krisstina Gowin (Mayo Clinical Arizona and Phoenix Children's Hospital) Ruben Mesa (Mayo Clinical Arizona and Phoenix Children's Hospital) Karen Ballen (University of Virgina Health System) Status: Submitted (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Mayo Clinic Arizona and Phoenix Children’s Hospital, Duke University Medical Center, Massachusetts General Hospital, M.D. Anderson Cancer Center, Cleveland Clinic Foundation, Dana-Farber Cancer Institute, Medical College of Wisconsin, Memorial Sloan Kettering Cancer Center, H. Lee Moffit Cancer Center and Research Institute, Northwestern University, University of Michigan, Washington University CK15-03

Title: Outcome of allogeneic hematopoietic cell transplantation in patients with acute myeloid leukemia with antecedent history of Philadelphia-negative myeloproliferative neoplasm

PI: Vikas Gupta (Princess Margaret Hospital) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) CK16-01 Title: Identification of germline predisposition mutations in young myelodysplastic

syndrome patients PI: Lucy Godley (University of Chicago Medicine) Status: Sample Typing (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with University of Chicago


Page | 20

CK16-02a Title: Contemporary role of maintenance tyrosine kinase inhibitors following

allogeneic hematopoietic cell transplantation for chronic myeloid leukemia: a CIBMTR analysis

PIs: Zach DeFilipp (Massachusetts General Hospital) Richard Ancheta (Scripps Blood & Marrow Transplant Program) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) CK16-02b Title: The benefit of donor lymphocyte infusion in the tyrosine kinase inhibitors era in

chronic myeloid leukemia post allogeneic hematopoietic cell transplantation PI: Sarah Schmidt (OU Medical Center and The Children's Hospital at OU Medical Center) Status: Submitted (as of July 1, 2019)


CK17-01 Title: Development of a prognostic scoring system predictive of outcomes in patients

with myelofibrosis after allogeneic hematopoietic cell transplantation PI: Roni Tamari (Memorial Sloan Kettering Cancer Center) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with the European Society for Blood and Marrow Transplantation (EBMT) CK17-02 Title: Reduced-intensity conditioning transplantation in older myelodysplastic

syndrome: the effect of specific conditioning regimens on transplant outcomes PI: Betul Oran (M.D. Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) CK18-01 Title: A personalized prediction model for outcomes after allogeneic stem cell

transplant in patients with myelodysplastic syndromes PIs: Aziz Nazha (Cleveland Clinic Foundation) Navneet Majhail (Cleveland Clinic Foundation) Wael Saber (Medical College of Wisconsin) Betty Hamilton (Cleveland Clinic Foundation) Status: Manuscript Preparation (as of July 1, 2019)



Page | 21

CK18-02 Title: The impact of somatic mutations on allogeneic transplant in chronic

myelomonocytic leukemia PIs: Matthew Mei (City of Hope) Ryotaro Nakamura (City of Hope) Raju Pillai (City of Hope) Status: Sample Typing (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) CK18-03 Title: Impact of donor age on the outcomes of allogeneic hematopoistic stem cell

transplantation for myelodysplastic syndrome PIs: Guru Murthy (Medical College of Wisconsin) Wael Saber (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)


CK19-01a Title: Outcomes after HCT for rare chronic leukemias: Evaluating outcomes of

Allogeneic hematopoietic cell transplantation in T-cell prolymphocytic leukemias PIs: Hemant Murthy (Mayo Clinic Florida) Bhagi Dholaria (Vanderbilt University Medical Center) Mohamed Kharfan (Mayo Clinic Florida) Susan Bal (University of Alabama at Birmingham) Craig Sauter (Memorial Sloan Kettering Cancer Center) Lohith Gowda (Yale New Haven Hospital) Francine Foss (Yale New Haven Hospital) Hassan Alkhateeb (Mayo Clinic Rochester) Deepa Jagadeesh (Cleveland Clinic Foundation) Bipin Savani (Vanderbilt University Medical Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) CK19-01b Title: Outcomes after HCT for rare chronic leukemias: Outcomes of chronic neutrophilic

leukemia patients who underwent alloHCT PIs: Bhagi Dholaria (Vanderbilt University Medical Center) Bipin Savani (Vanderbilt University Medical Center) Mohamed Kharfan (Mayo Clinic Florida) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with EBMT


Page | 22

SC11-06 / 10-CMSMDS-1 Title: Assessment of allogeneic hematopoietic stem cell transplantation in Medicare

beneficiaries with myelodysplastic syndrome and related disorders PIs: Ehab Atallah (Medical College of Wisconsin) JD Rizzo (Medical College of Wisconsin) Status: Submitted (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 5.0 DONOR HEALTH AND SAFETY WORKING COMMITTEE

Page | 23

5.0 DONOR HEALTH AND SAFETY WORKING COMMITTEE

5.1 Leadership

Chair: Nirali Shah, MD, MHSc, National Cancer Institute Email: [email protected] Chair: Galen Switzer, PhD, University of Pittsburgh Medical Center Email: [email protected] Chair: Jack Hsu, MD, Shands HealthCare & University of Florida Email: [email protected] Scientific Director: Bronwen Shaw, MBChB, MRCP, PhD, CIBMTR Milwaukee Email: [email protected] Ex Officio Sr Advisor: Dennis Confer, MD, CIBMTR Minneapolis Email: [email protected] Statistical Director: Brent Logan, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Stephanie Bo-Subait, MPH, CIBMTR Minneapolis Email: [email protected]


2019

Wiener L, Hoag JA, Pelletier W, Shah NN, Shaw BE, Pulsipher MA, Bruce J, Bader P, Willasch AM, Dalissier A, Guilcher G, Anthias C, Confer DL, Sees JA, Logan B, Switzer GE. Transplant center practices for psychosocial assessment and management of pediatric hematopoietic stem cell donors. Bone Marrow Transplantation. doi:10.1038/s41409-019-0515-3. Epub 2019 Apr 10. ncbi.nlm.nih.gov/pubmed/30971776

Prokopishyn NL, Logan BR, Kiefer DM, Sees JA, Chitphakdithai P, Ahmed IA, Anderlini PN, Beitinjaneh AM, Bredeson CN, Cerny J, Chhabra S, Daly A, Diaz MA, Farhadfar N, Frangoul HA, Ganguly S, Gastineau DA, Gergis U, Hale GA, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Murthy HS, Norkin M, Olsson RF, Papari M, Savani BN, Szer J, Waller EK, Wirk B, Yared JA, Pulsipher MA, Shah NN, Switzer GE, O'Donnell PV, Confer DL, Shaw BE. The concentration of total nucleated cells in harvested bone marrow for transplantation has decreased over time. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.01.034. Epub 2019 Feb 2. ncbi.nlm.nih.gov/pubmed/30716454











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2018

Pulsipher MA, Logan BR, Kiefer DM, Chitphakdithai P, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Varni JW, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Ball ED, Bolwell BJ, Bunin NJ, Cheerva A, Delgado DC, Dvorak CC, Gillio A, Hahn TE, Hale GA, Haight AE, Hayes-Lattin BM, Kasow KA, Linenberger M, Magalhaes-Silverman M, Mori S, Prasad VK, Quigg TC, Sahdev I, Schriber JR, Shenoy S, Tse WT, Yanik GA, Navarro WH, Horowitz MM, Confer DL, Shaw BE, Switzer GE. Higher risks of toxicity and incomplete recovery in 13-17 year old females after marrow donation: RDSafe peds results. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 May 1; 25(5):955-964. doi:10.1016/j.bbmt.2018.12.765. Epub 2018 Dec 31. PMC6511296. ncbi.nlm.nih.gov/pubmed/30605731

Pulsipher MA, Logan BR, Chitphakdithai P, Kiefer DM, Riches ML, Rizzo JD, Anderlini P, Leitman SF, Varni JW, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Akard LP, Artz AS, Ball ED, Bayer R-L, Bigelow C, Bolwell BJ, Broun ER, Bunin NJ, Delgado DC, Duckworth K, Dvorak CC, Hahn TE, Haight AE, Hari PN, Hayes-Lattin BM, Jacobsohn DA, Jakubowski AA, Kasow KA, Lazarus HM, Liesveld JL, Linenberger M, Litzow MR, Longo W, Magalhaes-Silverman M, McCarty JM, McGuirk JP, Mori S, Prasad VK, Rowley SD, Rybka WB, Sahdev I, Schriber JR, Selby GB, Shaughnessy PJ, Shenoy S, Spitzer T, Tse WT, Uberti JP, Vusirikala M, Waller EK, Weisdorf DJ, Yanik GA, Navarro WH, Horowitz MM, Switzer GE, Shaw BE, Confer DL. Effect of aging and predonation comorbidities on the related peripheral blood stem cell donor experience: Report from the Related Donor Safety Study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Apr 1; 25(4):699-711. doi:10.1016/j.bbmt.2018.11.004. Epub 2018 Nov 10. PMC6453753. ncbi.nlm.nih.gov/pubmed/30423480

Pulsipher MA, Logan BR, Kiefer DM, Chitphakdithai P, Riches ML, Rizzo JD, Anderlini P, Leitman OF, Kobusingye H, Besser RM, Miller JP, Drexler RJ, Abdel-Mageed A, Ahmed IA, Akard LP, Artz AS, Ball ED, Bayer R-L, Bigelow C, Bolwell BJ, Broun ER, Delgado DC, Duckworth K, Dvorak CC, Hahn TE, Haight AE, Hari PN, Hayes-Lattin BM, Jacobsohn DA, Jakubowski AA, Kasow KA, Lazarus HM, Liesveld JL, Linenberger M, Litzow MR, Longo W, Magalhaes-Silverman M, McCarty JM, McGuirk JP, Mori S, Parameswaran V, Prasad VK, Rowley SD, Rybka WB, Sahdev I, Schriber JR, Selby GB, Shaughnessy PJ, Shenoy S, Spitzer T, Tse WT, Uberti JP, Vusirikala M, Waller EK, Weisdorf DJ, Yanik GA, Navarro WH, Horowitz MM, Switzer GE, Confer DL, Shaw BE. Related peripheral blood stem cell donors experience more severe symptoms and less complete recovery at one year compared to unrelated donors. Haematologica. 2019 Apr 1; 104(4):844-854. doi:10.3324/haematol.2018.200121. Epub 2018 Oct 31. PMC6442962. ncbi.nlm.nih.gov/pubmed/30381298

Switzer GE, Macis M, Fabi R, Abress L, Confer D, Bruce J, Howe K, Fowler S, McNulty M, Pastorek G, Dew MA. Providing level-of-match information to perfectly matched unrelated donors: Evaluating acceptability and potential changes in donor availability. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.03.017. Epub 2018 Mar 21. ncbi.nlm.nih.gov/ pubmed/29574124







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2017

Stroncek DF, Shaw BE, Logan BR, Kiefer DM, Savani BN, Anderlini P, Bredeson CN, Hematti P, Ganguly S, Diaz MA, Abdel-Azim H, Ahmed I, Maharaj D, Seftel M, Beitinjaneh A, Seo S, Yared JA, Halter J, O'Donnell PV, Hale GA, DeFilipp Z, Lazarus H, Liesveld JL, Zhou Z, Munshi P, Olsson RF, Kasow KA, Szer J, Switzer GE, Chitphakdithai P, Shah N, Confer DL, Pulsipher MA. Donor experiences of second marrow or peripheral blood stem cell collection mirror the first, but CD34+ yields are less. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jan 1; 24(1):175-184. doi:10.1016/j.bbmt.2017.09.013. Epub 2017 Sep 25. PMC5743544. ncbi.nlm.nih.gov/ pubmed/28958894

Switzer GE, Bruce J, Pastorek G, Kiefer DM, Kobusingye H, Drexler R, Besser RAM, Confer DL, Horowitz MM, King RJ, Shaw BE, van Walraven SM, Weiner L, Packman W, Varni JW, Pulsipher MA. Parent versus child donor perceptions of the bone marrow donation experience. Bone Marrow Transplantation. 2017 Sep 1; 52(9):1338-1341. doi:10.1038/bmt.2017.124. Epub 2017 Jun 26. PMC5933883. ncbi.nlm.nih.gov/pubmed/28650449

2016

Switzer GE, Bruce J, Kiefer DM, Kobusingye H, Drexler R, Besser RM, Confer DL, Horowitz MM, King RJ, Shaw BE, Riches M, Hayes-Lattin B, Linenberger M, Bolwell B, Rowley SD, Litzow MR, Pulsipher MA. Health-related quality of life among older related hematopoietic stem cell donors (>60 years) is equivalent to that of younger related donors (18 to 60 years): A related donor safety study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):165-171. doi:10.1016/j.bbmt.2016.10.008. Epub 2016 Oct 14. PMC5182103. ncbi.nlm.nih.gov/ pubmed/27751935

Switzer GE, Bruce J, Kiefer DM, Kobusingye H, Drexler R, Besser RM, Confer DL, Horowitz MM, King RJ, Shaw BE, van Walraven SM, Wiener L, Packman W, Varni JW, Pulsipher MA. Health-related quality of life among pediatric hematopoietic stem cell donors. The Journal of Pediatrics. 2016 Nov 1; 178:164-170.e1. doi:10.1016/j.jpeds.2016.07.009. Epub 2016 Aug 10. PMC5085860. ncbi.nlm.nih.gov/pubmed/27522440

2015

Anthias C, O'Donnell PV, Kiefer DM, Yared J, Norkin M, Anderlini P, Savani BN, Diaz MA, Bitan M, Halter JP, Logan BR, Switzer GE, Pulsipher MA, Confer DL, Shaw BE. European Group for Blood and Marrow Transplantation centers with FACT-JACIE accreditation have significantly better compliance with related donor care standards. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Mar 1; 22(3):514-519. doi:10.1016/j.bbmt.2015.11.009. Epub 2015 Nov 18. PMC4760354. ncbi.nlm.nih.gov/pubmed/26597079









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Anthias C, Shaw BE, Kiefer DM, Liesveld JL, Yared J, Kamble RT, D'Souza A, Hematti P, Seftel MD, Norkin M, DeFilipp Z, Kasow KA, Abidi MH, Savani BN, Shah NN, Anderlini P, Diaz MA, Malone AK, Halter JP, Lazarus HM, Logan BR, Switzer GE, Pulsipher MA, Confer DL, O'Donnell PV. Significant improvements in the practice patterns of adult related donor care in US transplantation centers. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Mar 1; 22(3):520-527. doi:10.1016/j.bbmt.2015. 11.008. Epub 2015 Nov 18. PMC4760616. ncbi.nlm.nih.gov/pubmed/26597080

Shaw BE, Logan BR, Kiefer DM, Chitphakdithai P, Pedersen TL, Abdel-Azim H, Abidi MH, Akpek G, Diaz MA, Artz AS, Dandoy C, Gajewski JL, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Majhail NS, O'Donnell PV, Olsson RF, Savani BN, Schears RM, Stroncek DF, Switzer GE, Williams EP, Wingard JR, Wirk BM, Confer DL, Pulsipher MA. Analysis of the effect of race, socioeconomic status, and center size on unrelated National Marrow Donor Program donor outcomes: donor toxicities are more common at low-volume bone marrow collection centers. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1830-1838. doi:10.1016/ j.bbmt.2015.06.013. Epub 2015 Jun 23. PMC4568129. ncbi.nlm.nih.gov/pubmed/26116089

2014

Hsu JW, Wingard JR, Logan BR, Chitphakdithai P, Akpek G, Anderlini P, Artz AS, Bredeson C, Goldstein S, Hale G, Hematti P, Joshi S, Kamble RT, Lazarus HM, O'Donnell PV, Pulsipher MA, Savani BN, Schears RM, Shaw BE, Confer DL. Race and ethnicity influences collection of granulocyte colony-stimulating factor-mobilized peripheral blood progenitor cells from unrelated donors, a Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):165-171. doi:10.1016/j.bbmt.2014. 10.007. Epub 2014 Oct 12. PMC4272878. ncbi.nlm.nih.gov/pubmed/25316111

5.3 Current Studies

DS05-02d Title: QoL for related adult donors compared to unrelated adult donors PIs: Galen Switzer (University of Pittsburgh Medical Center) Michael Pulsipher (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with University of Utah DS05-02g Title: Late toxicities and SAE for related donors PI: Michael Pulsipher (Children's Hospital of Los Angeles) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with University of Utah





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DS13-02 Title: A retrospective analysis to understand the potential mechanisms underlying the

clinical impact of ABO incompatibility on allogeneic transplant outcomes PI: Guru Murthy (Medical College of Wisconsin) Status: Protocol Development (as of July 1, 2019)

Analysis (expected by June 30, 2020)

DS16-01 Title: Comparison between one and two day apheresis in unrelated donors PIs: Jack W. Hsu (Shands HealthCare & University of Florida) John W. Wingard (Shands HealthCare & University of Florida) Status: Manuscript Preparation (as of July 1, 2019)


DS16-S2 Title: Survey of the screening and management for clonal disorders of hematopoiesis in

related allogeneic donors PI: Matthew Seftel (CancerCare Manitoba / Univeristy of Manitoba) Status: Submitted (as of July 1, 2019)


DS17-01 Title: The impact of donor body mass index on collection of G-CSF mobilized peripheral

blood progenotor cells from unrelated donors PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) Jack Hsu (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Status: Submitted (as of July 1, 2019)


DS17-02 Title: Impact of pre-operative collection of auto blood for BM harvest on donor health

and outcome PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Hemant Murthy (Mayo Clinic Florida) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with American Association of Blood Banks (AABB) / American Society for

Transplantation and Cellular Therapy (ASTCT)


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DS18-01 Title: To quantify age-related clonal hematopoiesis in healthy marrow and blood

donors that may affect the clinical outcome of recipients following HCT PI: Todd Druley (Washington University School of Medicine) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with Washington University

DS18-02 Title: Factors affecting CD34+ cell yields at subsequent marrow/PBSC collections. PIs: Sandhya Panch (National Institutes of Health Clinical Center) David Stronecek (National Heart, Lung and, Blood Institute) Bipin Savani (Vanderbilt University Medical Center) Nirali N. Shah (National Institutes of Health) Status: Manuscript Preparation (as of July 1, 2019)


DS19-01 Title: Effect of Donor Graft Cryopreservation on Allogeneic Transplant Recipient

Outcomes PIs: Jack Hsu (Shands HealthCare & University of Florida) Nosha Farhadfar (Shands HealthCare & University of Florida) Hemant Murthy (Mayo Clinic Florida) John Wingard (Shands HealthCare & University of Florida) Status: Draft Protocol Received (as of July 1, 2019)


DS19-02 Title: The Impact of pre-apheresis Health related quality of life on peripheral blood

progenitor cells yield and donor’s health and outcome PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) John Wingard (Shands HealthCare & University of Florida) Galen Switzer (University of Pittsburgh Medical Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with University of Pittsburgh

2019 CIBMTR Scientific Working Committee Research Portfolio 6.0 GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE

Page | 29

6.0 GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE

6.1 Leadership

Chair: Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside Hospital

Email: [email protected] Chair: Ian McNiece, PhD, University of Miami, CellMED Consulting Email: [email protected] Chair: Claudio Brunstein, MD, PhD, University of Minnesota Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Mei-Jie Zhang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Mariam Johnson, MPH, CIBMTR Milwaukee Email: [email protected]


2019

Perales M-A, Tomlinson B, Zhang M-J, St. Martin A, Beitinjaneh A, Gibson J, Hogan W, Kekre N, Lazarus H, Marks D, McGuirk J, Romee R, Solh M, Wagner JE, Weisdorf DJ, de Lima M, Eapen M. Alternative donor transplantation for acute myeloid leukemia in patients aged ≥50 years: Young HLA-matched unrelated or haploidentical donor? Haematologica. doi:10.3324/ haematol.2018.215202. Epub 2019 May 17. ncbi.nlm.nih.gov/pubmed/31101756

2018

Keesler DA, St. Martin A, Bonfim C, Seber A, Zhang M-J, Eapen M. Bone marrow versus peripheral blood from unrelated donors for children and adolescents with acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Dec 1; 24(12):2487-2492. doi:10.1016/j.bbmt. 2018.08.010. Epub 2018 Aug 21. PMC6286246. ncbi.nlm.nih.gov/pubmed/30142417

Robinson TM, Fuchs EJ, Zhang M-J, St Martin A, Labopin M, Keesler DA, Blaise D, Bashey A, Bourhis J-H, Ciceri F, Ciurea SO, Devine SM, Mohty M, McCurdy SR, Milpied N, McNiece IK, Rocha V, Romee R, Socie G, Yakoub-Agha I, Soiffer RJ, Eapen M, Nagler A. Related donor transplants: Has posttransplantation cyclophosphamide nullified the detrimental effect of HLA mismatch? Blood Advances. 2018 Jun 12; 2(11):1180-1186. doi:10.1182/bloodadvances. 2018018291. Epub 2018 May 24. ncbi.nlm.nih.gov/pubmed/29794073











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Stiff PJ, Montesinos P, Peled T, Landau E, Rosenheimer Goudsmid N, Mandel J, Hasson N, Olesinski E, Glukhman E, Snyder DA, Galamidi Cohen E, Srur Kidron O, Bracha D, Harati D, Ben-Abu K, Freind E, Freedman LS, Cohen YC, Olmer L, Barishev R, Rocha V, Gluckman E, Horowitz MM, Eapen M, Nagler A, Sanz G. Cohort-controlled comparison of umbilical cord blood transplantation using carlecortemcel-L, a single progenitor-enriched cord blood, to double cord blood unit transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2018.02.012. Epub 2018 Mar 1. ncbi.nlm.nih.gov/pubmed/29477778

McCurdy SR, Zhang M-J, St Martin A, Al Malki MM, Bashey A, Gaballa S, Keesler DA, Hamadani M, Norkin M, Perales M-A, Reshef R, Rocha V, Romee R, Solh M, Urbano-Ispizua A, Waller EK, Fuchs EJ, Eapen M. Effect of donor characteristics on haploidentical transplantation with posttransplantation cyclophosphamide. Blood Advances. 2018 Feb 13; 2(3):299-307. doi:10.1182/bloodadvances.2017014829. Epub 2018 Feb 9. PMC5812334. ncbi.nlm.nih.gov/ pubmed/29440002

2017

Bashey A, Zhang M-J, McCurdy SR, St, Martin A, Argall T, Anasetti C, Ciurea SO, Fasan O, Gaballa S, Hamadani M, Munshi P, Al Malki MM, Nakamura R, O'Donnell PV, Perales M-A, Raj K, Romee R, Rowley S, Rocha V, Salit RB, Solh M, Soiffer RJ, Fuchs EJ, Eapen M. Mobilized peripheral blood stem cells versus unstimulated bone marrow as a graft source for T-cell replete haploidentical donor transplantation using post-transplant cyclophosphamide. Journal of Clinical Oncology. 2017 Sep 10; 35(26):3002-3009. doi:10.1200/JCO.2017.72.8428. Epub 2017 Jun 23. PMC5590802. ncbi.nlm.nih.gov/pubmed/28644773

Armstrong AE, Fonstad R, Spellman S, Tullius Z, Chaudhury S. Current knowledge and practice of pediatric providers in umbilical cord blood banking. Clinical Pediatrics. 2018 Feb 1; 57(2):161-167. doi:10.1177/0009922817692316. Epub 2017 Mar 1. ncbi.nlm.nih.gov/pubmed/ 28952356

Brunstein C, Zhang M-J, Barker J, St, Martin A, Bashey A, de Lima M, Dehn J, Hematti P, Perales MA, Rocha V, Territo M, Weisdorf D, Eapen M. The effect of inter-unit HLA matching in double umbilical cord blood transplantation for acute leukemia. Haematologica. 2017 May 1; 102(5):941-947. doi:10.3324/haematol.2016.158584. Epub 2017 Jan 25. PMC5477613. ncbi.nlm.nih.gov/pubmed/28126967

2016

Brady C, Armitage S, Freed B, Duffy M, Gass A, Spellman S, Kurtzberg J, Regan D. How transplant centers deal with the dextran shortage: Recommendations for comparing alternatives. Transfusion. 2016 Nov 1; 56(11):2657-2661. doi:10.1111/trf.13783. Epub 2016 Sep 5. ncbi.nlm.nih.gov/pubmed/27596242

Kim HT, Zhang M-J, Woolfrey AE, St, Martin A, Chen J, Saber W, Perales M-A, Armand P, Eapen M. Donor and recipient sex in allogeneic stem cell transplantation: What really matters. Haematologica. 2016 Oct 1; 101(10):1260-1266. doi:10.3324/haematol.2016.147645. Epub 2016 Jun 27. PMC5046656. ncbi.nlm.nih.gov/pubmed/27354023











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Rafii H, Ruggeri A, Volt F, Brunstein CG, Carreras J, Eapen M, Gluckman E, Weisdorf DJ. Changing trends of unrelated umbilical cord blood transplantation for hematologic diseases in patients older than fifty years: A Eurocord-Center for International Blood and Marrow Transplant research survey. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Sep 1; 22(9):1717-1720. doi:10.1016/j.bbmt.2016.05.015. Epub 2016 May 21. ncbi.nlm.nih.gov/pubmed/27220265

Van Besien K, Hari P, Zhang M-J, Liu H-T, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival. Haematologica. 2016 May 1; 101(5):634-643. doi:10.3324/haematol.2015.138594. Epub 2016 Feb 11. PMC5004373. ncbi.nlm.nih.gov/pubmed/26869630

Al Malki MM, Horowitz M, Handgretinger R, Leung W, Roy D-C, Huang X-J, Fuchs E, Locatelli F, Blaise D, Mineishi S, Martelli M, Miller J, June C, Ai H-S, Luznik L, Mavilio D, Lugli E, van den Brink MRM, Champlin RE, Ciurea SO. Proceedings from the Second Haploidentical Stem Cell Transplantation Symposium - Haplo2014, San Francisco, California, December 4, 2014. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):594-604. doi:10.1016/j.bbmt.2016.01.001. Epub 2016 Jan 13. ncbi.nlm.nih.gov/pubmed/26806585

2015

Kollman C, Spellman SR, Zhang M-J, Hassebroek A, Anasetti C, Antin JH, Champlin RE, Confer DL, DiPersio JF, Fernandez-Viña M, Hartzman RJ, Horowitz MM, Hurley CK, Karanes C, Maiers M, Mueller CR, Perales M-A, Setterholm M, Woolfrey AE, Yu N, Eapen M. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood. 2016 Jan 14; 127(2):260-267. doi:10.1182/blood-2015-08-663823. Epub 2015 Nov 2. PMC4713163. ncbi.nlm.nih.gov/pubmed/26527675

Ponce DM, Eapen M, Sparapani R, O'Brien TA, Chan KW, Chen J, Craddock J, Schultz KR, Wagner JE, Perales M-A, Barker JN. In vivo T cell depletion with myeloablative regimens on outcomes after cord blood transplantation for acute lymphoblastic leukemia in children. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2173-2179. doi:10.1016/j.bbmt.2015.08.022. Epub 2015 Aug 29. PMC4639413. ncbi.nlm.nih.gov/pubmed/26327630

Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales M-A, Pingali SR, Porter DL, Riches MR, Ringdén OTH, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M. Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. 2015 Aug 20; 126(8):1033-1040. doi:10.1182/blood-2015-04-639831. Epub 2015 Jun 30. PMC4543223. ncbi.nlm.nih.gov/pubmed/26130705








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2014

Ballen KK, Logan BR, Laughlin MJ, He W, Ambruso DR, Armitage SE, Beddard RL, Bhatla D, Hwang WYK, Kiss JE, Koegler G, Kurtzberg J, Nagler A, Oh D, Petz LD, Price TH, Quinones RR, Ratanatharathorn V, Rizzo JD, Sazama K, Scaradavou A, Schuster MW, Sender LS, Shpall EJ, Spellman SR, Sutton M, Weitekamp LA, Wingard JR, Eapen M. Effect of cord blood processing on transplantation outcomes after single myeloablative umbilical cord blood transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):688-695. doi:10.1016/j.bbmt.2014. 12.017. Epub 2014 Dec 24. PMC4359657. ncbi.nlm.nih.gov/pubmed/25543094

Eapen M, Logan BR, Horowitz MM, Zhong X, Perales M-A, Lee SJ, Rocha V, Soiffer RJ, Champlin RE. Bone marrow or peripheral blood for reduced-intensity conditioning unrelated donor transplantation. Journal of Clinical Oncology. 2015 Feb 1; 33(4):364-369. doi:10.1200/JCO. 2014.57.2446. Epub 2014 Dec 22. PMC4302216. ncbi.nlm.nih.gov/pubmed/25534391

Eapen M, Logan BR, Appelbaum FR, Antin JH, Anasetti C, Couriel DR, Chen J, Maziarz RT, McCarthy PL, Nakamura R, Ratanatharathorn V, Vij R, Champlin RE. Long-term survival after transplantation of unrelated donor peripheral blood or bone marrow hematopoietic cells for hematologic malignancy. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):55-59. doi:10.1016/ j.bbmt.2014.09.006. Epub 2014 Sep 22. PMC4272909. ncbi.nlm.nih.gov/pubmed/25255165

6.3 Current Studies

GS18-01 Title: Mismatch in the setting of PT-CY based anti-GVHD prophylaxis: Is a matched

related, matched unrelated or haploidentical donor still an issue? / Use of post-transplant cyclophosphamide in alloHCT: HLA mismatched donors vs haplo-identical donors vs fully matched unrelated donor vs fully matched related donors

PIs: Saurabh Chhabra (Medical College of Wisconsin) Kehinde Adekola (Northwestern Medicine) Mahasweta Gooptu (Thomas Jefferson University Hospital, Inc.) Miguel-Angel Perales (Memorial Sloan Kettering Cancer Center) Alberto Mussetti (Instituto Nazionale Tumori) Rizwan Romee (Dana Farber Cancer Institute at Brigham and Women's Hospital) Status: Draft Protocol Received (as of July 1, 2019)






Page | 33

GS18-02 Title: Impact of racial background on survival following haploidentical donor

transplantation with post-transplant cyclophosphamide for adults with hematologic malignancies and comparison with race-specific outcomes following umbilical cord blood transplantation

PIs: Scott Solomon (The Blood and Marrow Transplant Program at Northside Hospital) Asad Bashey (The Blood and Marrow Transplant Program at Northside Hospital) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) GS18-03 Title: Comparison of outcomes of reduced intensity transplantation in lymphoma

patients using haploidentical related donors vs. unrelated cord blood PIs: Giancarlo Fatobene (Seattle Cancer Care Alliance) Vanderson Rocha (Churchill Hospital) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with EBMT GS18-04 Title: Comparison of outcomes with haploidentical and matched unrelated donors for

hematopoietic stem cell transplantation in myelodysplastic syndromes / Outcomes of HLA-haploidentical allogeneic blood or marrow transplantation with post-transplant cyclophosphamide for myeloproliferatice neoplasms and myelodysplastic sydrome / myelowproliferatice neoplasm overlap syndromes

PIs: Auro Viswabandya (Princess Margaret Cancer Center - BMT Program) Benjamin Tomlinson (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Michael Grunwald (Levine Cancer Institute) Hany Elmariah (Johns Hopkins University) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) GS19-01 Title: Comparison of myeloablative haploidentical or umbilical cord blood

transplatation for pediatric and adult patients with acute leukemia PIs: John Wagner (University of Minnesota Blood and Marrow Transplant Program) Karen Ballen (University of Virginia Health System) Status: Data File Preparation (as of July 1, 2019)



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GS19-02 Title: Graft failure in MDS and acute leukemia patients after allogeneic stem cell

transplantation receiving post transplant cyclophosphamide PIs: Cindy Lynn Hickey-Gillis (Dana Farber Cancer Institute) Rizwan Romee (Dana Farber Cancer Institute at Brigham and Women's Hospital) Corey Cutler (Dana Farber Cancer Institute at Brigham and Women's Hospital) Navneet Majhail (Cleveland Clinic Foundation) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) GS19-03 Title: Impact of G-CSF on in-vivo T-cell depleted allogeneic hematopoietic cell

transplantation PIs: Nina Orfali (Weil Cornell Medicine) Jaap Jan Boelens (Memorial Sloan Kettering Cancer Center) Koen Van Besien (New York Presbyterian Hospital at Cornell) Status: Draft Protocol Received (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 7.0 GRAFT-VS-HOST DISEASE WORKING COMMITTEE

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7.0 GRAFT-VS-HOST DISEASE WORKING COMMITTEE

7.1 Leadership

Chair: Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute

Email: [email protected] Chair: Madan Jagasia, MBBS, MS, Vanderbilt University Medical Center Email: [email protected] Chair: Margaret MacMillan, MD, MSc, University of Minnesota Blood and

Marrow Transplant Program – Pediatrics Email: [email protected] Scientific Director: Mukta Arora, MD, MS, CIBMTR Minneapolis Email: [email protected] Scientific Director: Stephen Spellman, MBS, CIBMTR Minneapolis Email: [email protected] Statistical Director: Tao Wang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Karen Chen , MS, CIBMTR Milwaukee Email: [email protected]


2019

Hamilton BK, Liu Y, Hemmer MT, Majhail N, Ringden O, Kim D, Costa L, Stuart R, Alousi A, Pidala JA, Couriel DR, Aljurf M, Antin JH, Bredeson C, Cahn J-Y, Cairo M, Choi SW, Dandoy C, Gale RP, Gergis U, Hematti P, Inamoto Y, Kamble RT, MacMillan M, Marks DI, Nemecek E, Nishihori T, Saad A, Savani BN, Schriber J, Seo S, Socié G, Teshima T, Verdonck LF, Waller EK, Wirk M, Spellman SR, Arora M, Chhabra S. Inferior outcomes with cyclosporine and mycophenolate mofetil after myeloablative allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.05.019. Epub 2019 May 31. ncbi.nlm.nih.gov/pubmed/31158502

Saad A, Lamb L, Wang T, Hemmer MT, Spellman S, Couriel D, Alousi A, Pidala J, Abdel-Azim H, Agrawal V, Aljurf M, Beitinjaneh AM, Bhatt VR, Buchbinder D, Byrne M, Cahn J-Y, Cairo M, Castillo P, Chhabra S, Diaz MA, Farhan S, Floisand Y, Frangoul HA, Gadalla SM, Gajewski J, Gale RP, Gandhi M, Gergis U, Hamilton BK, Hematti P, Hildebrandt GC, Kamble RT, Kanate AS, Khandelwal P, Lazaryan A, MacMillan M, Marks DI, Martino R, Mehta PA, Nishihori T, Olsson RF, Patel SS, Qayed M, Rangarajan H, Reshef R, Ringden O, Savani BN, Schouten HC, Schultz KR, Seo S, Shaffer BC, Solh M, Teshima T, Urbano-Ispizua A, Verdonck LF, Vij R, Waller EK, William B, Wirk B, Yared J, Yu LC, Arora M, Hashmi S. Impact of T-cell dose on the outcome of T-cell replete HLA matched allogeneic peripheral blood stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American










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Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.05.007. Epub 2019 May 11. ncbi.nlm.nih.gov/pubmed/31085303

Mehta RS, Holtan SG, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel DR, Alousi AM, Pidala J, Abdel-Azim H, Ahmed I, Aljurf M, Askar M, Auletta JJ, Bhatt V, Bredeson C, Chhabra S, Gadalla S, Gajewski J, Gale RP, Gergis U, Hematti P, Hildebrandt GC, Inamoto Y, Kitko C, Khandelwal P, MacMillan ML, Majhail N, Marks DI, Mehta P, Nishihori T, Olsson RF, Pawarode A, Diaz MA, Prestidge T, Qayed M, Rangarajan H, Ringden O, Saad A, Savani BN, Seo S, Shah A, Shah N, Schultz KR, Solh M, Spitzer T, Szer J, Teshima T, Verdonck LF, Williams KM, Wirk B, Wagner J, Yared JA, Weisdorf DJ. GRFS and CRFS in alternative donor hematopoietic cell transplantation for pediatric patients with acute leukemia. Blood Advances. 2019 May 14; 3(9):1441-1449. doi:10.1182/bloodadvances.2018030171. Epub 2019 May 3. PMC6517657. ncbi.nlm.nih.gov/pubmed/31053571

2018

Alousi A, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel DR, Pidala J, Anderlini P, Boyiadzis M, Bredeson CN, Cahn J-Y, Cairo MS, Gadalla SM, Hashmi SK, Gale RP, Kanda J, Kamble RT, Kharfan-Dabaja MA, Litzow MR, Ringden O, Saad AA, Schultz KR, Verdonck LF, Waller EK, Yared JA, Holtan SG, Weisdorf DJ. Peripheral blood versus bone marrow from unrelated donors: Bone marrow allografts have improved long-term overall and graft-versus-host disease-free, relapse-free survival. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Feb 1; 25(2):270-278. doi:10.1016/j.bbmt.2018.09.004. Epub 2018 Oct 3. PMC6339839. ncbi.nlm.nih.gov/pubmed/30292009

Chhabra S, Liu Y, Hemmer MT, Costa L, Pidala JA, Couriel DR, Alousi AM, Majhail NS, Stuart RK, Kim D, Ringden O, Urbano-Ispizua A, Saad A, Savani BN, Cooper B, Marks DI, Socie G, Schouten HC, Schoemans H, Abdel-Azim H, Yared J, Cahn J-Y, Wagner J, Antin JH, Verdonck LF, Lehmann L, Aljurf MD, MacMillan ML, Litzow MR, Solh MM, Qayed M, Hematti P, Kamble RT, Vij R, Hayashi RJ, Gale RP, Martino R, Seo S, Hashmi SK, Nishihori T, Teshima T, Gergis U, Inamoto Y, Spellman SR, Arora M, Hamilton BK. Comparative analysis of calcineurin inhibitor-based methotrexate and mycophenolate mofetil-containing regimens for prevention of graft-versus-host disease after reduced-intensity conditioning allogeneic transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):73-85. doi:10.1016/j.bbmt.2018.08.018. Epub 2018 Aug 25. PMC6355336. ncbi.nlm.nih.gov/pubmed/30153491

Turcotte LM, Wang T, Hemmer MT, Spellman SR, Arora M, Yingst A, Couriel D, Alousi A, Pidala J, Knight JM, Verneris MR. Pro-inflammatory cytokine and adipokine levels in adult unrelated marrow donors are not associated with hematopoietic cell transplantation outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):12-18. doi:10.1016/j.bbmt.2018.08.011. Epub 2018 Aug 23. PMC6366664. ncbi.nlm.nih.gov/pubmed/30144561







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Nikiforow S, Wang T, Hemmer M, Spellman S, Akpek G, Antin JH, Choi SW, Inamoto Y, Khoury HJ, MacMillan M, Marks DI, Meehan K, Nakasone H, Nishihori T, Olsson R, Paczesny S, Przepiorka D, Reddy V, Reshef R, Schoemans H, Waller N, Weisdorf D, Wirk B, Horowitz M, Alousi A, Couriel D, Pidala J, Arora M, Cutler C. Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value in isolation or with other graft-versus-host disease symptoms as currently diagnosed and treated. Haematologica. doi:10.3324/haematol.2017.182550. Epub 2018 Aug 3. 30076185. ncbi.nlm.nih.gov/ pubmed/30076185

Schoemans HM, Lee SJ, Ferrara JL, Wolff D, Levine JE, Schultz KR, Shaw BE, Flowers ME, Ruutu T, Greinix H, Holler E, Basak G, Duarte RF, Pavletic SZ. EBMT-NIH-CIBMTR Task Force position statement on standardized terminology & guidance for graft-versus-host disease assessment. Bone Marrow Transplantation. doi:10.1038/s41409-018-0204-7. Epub 2018 Jun 5. ncbi.nlm.nih.gov/pubmed/29872128

Kumar AJ, Kim S, Hemmer MT, Arora M, Spellman SR, Pidala JA, Couriel DR, Alousi AM, Aljurf MD, Cahn J-Y, Cairo MS, Cutler CS, Farhan S, Gergis U, Hale GA, Hashmi SK, Inamoto Y, Kamble RT, Kharfan-Dabaja MA, MacMillan ML, Marks DI, Nakasone H, Norkin M, Qayed M, Ringden O, Schouten HC, Schultz KR, Solh MM, Teshima T, Urbano-Ispizua A, Verdonck LF, Gale RP, Hamilton BK, Majhail NS, Loren AW. Graft-versus-host disease in recipients of male unrelated donor compared with parous female sibling donor transplants. Blood Advances. 2018 May 8; 2(9):1022-1031. doi:10.1182/bloodadvances.2017013052. Epub 2018 May 8. PMC5941995. ncbi.nlm.nih.gov/pubmed/29739773

Turcotte LM, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel D, Alousi A, Pidala J, Abdel-Azim H, Ahmed I, Beitinjaneh A, Buchbinder D, Byrne M, Callander N, Chao N, Choi SW, DeFilipp Z, Gadalla SM, Gale RP, Gergis U, Hashmi S, Hematti P, Holmberg L, Inamoto Y, Kamble RT, Lehmann L, MacMillan MA, McIver Z, Nishihori T, Norkin M, O'Brien T, Olsson RF, Reshef R, Saad A, Savani B, Schouten HC, Seo S, Solh M, Verdonck L, Vij R, Wirk B, Yared J, Horowitz MM, Knight JM, Verneris MR. Donor body mass index does not predict graft versus host disease following hematopoietic cell transplantation. Bone Marrow Transplantation. doi:10.1038/s41409-018-0100-1. Epub 2018 Jan 30. ncbi.nlm.nih.gov/ pubmed/29382954

Drobyski WR, Szabo A, Zhu F, Keever-Taylor C, Hebert KM, Dunn R, Yim S, Johnson B, D'Souza A, Eapen M, Fenske TS, Hari P, Hamadani M, Horowitz MM, Rizzo JD, Saber W, Shah N, Shaw B, Pasquini M. Tocilizumab, tacrolimus and methotrexate for the prevention of acute graft-versus-host disease: Low incidence of lower gastrointestinal tract disease. Haematologica. 2018 Apr 1;103(4):717-727. doi:10.3324/haematol.2017.183434. Epub 2018 Jan 19. PMC5865423. ncbi.nlm.nih.gov/pubmed/29351985









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2017

Qayed M, Wang T, Hemmer MT, Spellman S, Arora M, Couriel D, Alousi A, Pidala J, Abdel-Azim H, Aljurf M, Ayas M, Bitan M, Cairo M, Choi SW, Dandoy C, Delgado D, Gale RP, Hale G, Frangoul H, Kamble RT, Kharfan-Dabaja M, Lehman L, Levine J, MacMillan M, Marks DI, Nishihori T, Olsson RF, Hematti P, Ringden O, Saad A, Satwani P, Savani BN, Schultz KR, Seo S, Shenoy S, Waller EK, Yu L, Horowitz MM, Horan J. Influence of age on acute and chronic GVHD in children undergoing HLA-identical sibling bone marrow transplantation for acute leukemia: Implications for prophylaxis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Mar 1; 24(3):521-528. doi:10.1016/j.bbmt.2017.11.004. Epub 2017 Nov 16. PMC5826854. ncbi.nlm.nih.gov/pubmed/ 29155316

Krakow EF, Hemmer M, Wang T, Logan B, Arora M, Spellman S, Couriel D, Alousi A, Pidala J, Last M, Lachance S, Moodie EEM. Tools for the precision medicine era: How to develop highly personalized treatment recommendations from cohort and registry data using Q-learning. American Journal of Epidemiology. 2017 Jul 15; 186(2):160-172. doi:10.1093/aje/kwx027. Epub 2017 May 5. ncbi.nlm.nih.gov/pubmed/28472335

Khoury HJ, Wang T, Hemmer MT, Couriel D, Alousi A, Cutler C, Aljurf M, Antin JH, Ayas M, Battiwalla M, Cahn J-Y, Cairo M, Chen Y-B, Gale RP, Hashmi S, Hayashi RJ, Jagasia M, Juckett M, Kamble RT, Kharfan Dabaja M, Litzow M, Majhail N, Miller A, Nishihori T, Qayed M, Schoemans H, Schouten HC, Socié G, Storek J, Verdonck L, Vij R, Wood WA, Yu L, Martino R, Carabasi M, Dandoy C, Gergis U, Hermatti P, Solh M, Jamani K, Lehmann L, Savani B, Schultz KR, Wirk BM, Spellman S, Arora M, Pidala J. Improved survival after acute graft vs. host disease diagnosis in the modern era. Haematologica. 2017 May 1; 102(5):958-966. doi:10.3324/haematol.2016.156356. Epub 2017 Mar 16. PMC5477615. ncbi.nlm.nih.gov/pubmed/28302712

2016

Chen Y-B, Wang T, Hemmer MT, Brady C, Couriel DR, Alousi A, Pidala J, Urbano-Isizua A, Choi SW, Nishihori T, Teshima T, Inamoto Y, Wirk B, Marks DI, Abdel-Azim H, Lehmann L, Yu L, Bitan M, Cairo MS, Qayed M, Salit R, Gale RP, Martino R, Jaglowski S, Bajel A, Savani B, Frangoul H, Lewis ID, Storek J, Askar M, Kharfan-Dabaja MA, Aljurf M, Ringden O, Reshef R, Olsson RF, Hashmi S, Seo S, Spitzer TR, MacMillan ML, Lazaryan A, Spellman SR, Arora M, Cutler CS. GVHD after umbilical cord blood transplantation for acute leukemia: An analysis of risk factors and effect on outcomes. Bone Marrow Transplantation. 2017 Mar 1; 52(3):400-408. doi:10.1038/bmt.2016.265. Epub 2016 Dec 12. PMC5332289. ncbi.nlm.nih.gov/pubmed/27941764







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2015

Inamoto Y, Flowers MED, Wang T, Urbano-Ispizua A, Hemmer MT, Cutler CS, Couriel DR, Alousi AM, Antin JH, Gale RP, Gupta V, Hamilton BK, Kharfan-Dabaja MA, Marks DI, Ringdén OTH, Socié G, Solh MM, Akpek G, Cairo MS, Chao NJ, Hayashi RJ, Nishihori T, Reshef R, Saad A, Shah A, Teshima T, Tallman MS, Wirk B, Spellman SR, Arora M, Martin PJ. Tacrolimus versus cyclosporine after hematopoietic cell transplantation for acquired aplastic anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1776-1782. doi:10.1016/j.bbmt. 2015.05.023. Epub 2015 May 30. PMC4568149. ncbi.nlm.nih.gov/pubmed/26033280

2014

Arora M, Hemmer MT, Ahn KW, Klein JP, Cutler CS, Urbano-Ispizua A, Couriel DR, Alousi AM, Gale RP, Inamoto Y, Weisdorf DJ, Li P, Antin JH, Bolwell BJ, Boyiadzis M, Cahn J-Y, Cairo MS, Isola LM, Jacobsohn DA, Jagasia M, Klumpp TR, Petersdorf EW, Santarone S, Schouten HC, Wingard JR, Spellman SR, Pavletic SZ, Lee SJ, Horowitz MM, Flowers ME. Center for International Blood and Marrow Transplant Research chronic graft-versus-host disease risk score predicts mortality in an independent validation cohort. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):640-645. doi:10.1016/j.bbmt.2014.10.022. Epub 2014 Dec 18. PMC4359642. ncbi.nlm.nih.gov/pubmed/25528390

Arai S, Arora M, Wang T, Spellman SR, He W, Couriel DR, Urbano-Ispizua A, Cutler CS, Bacigalupo AA, Battiwalla M, Flowers ME, Juckett MB, Lee SJ, Loren AW, Klumpp TR, Prockup SE, Ringdén OT, Savani BN, Socié G, Schultz KR, Spitzer T, Teshima T, Bredeson CN, Jacobsohn DA, Hayashi RJ, Drobyski WR, Frangoul HA, Akpek G, Ho VT, Lewis VA, Gale RP, Koreth J, Chao NJ, Aljurf MD, Cooper BW, Laughlin MJ, Hsu JW, Hematti P, Verdonck LF, Solh MM, Norkin M, Reddy V, Martino R, Gadalla S, Goldberg JD, McCarthy PL, Pérez-Simón JA, Khera N, Lewis ID, Atsuta Y, Olsson RF, Saber W, Waller EK, Blaise D, Pidala JA, Martin PJ, Satwani P, Bornhäuser M, Inamoto Y, Weisdorf DJ, Horowitz MM, Pavletic SZ. Increasing incidence of chronic graft-versus-host disease in allogeneic transplantation: A report from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):266-274. doi:10.1016/j.bbmt.2014.10.021. Epub 2014 Oct 30. PMC4326247. ncbi.nlm.nih.gov/pubmed/25445023

Boyiadzis M, Arora M, Klein JP, Hassebroek A, Hemmer M, Urbano-Ispizua A, Antin JH, Bolwell BJ, Cahn J-YY, Cairo MS, Cutler CS, Flowers ME, Gale RP, Herzig R, Isola LM, Jacobsohn DA, Jagasia MH, Klumpp TR, Lee SJ, Petersdorf EW, Santarone S, Spellman SR, Schouten HC, Verdonck LF, Wingard JR, Weisdorf DJ, Horowitz MM, Pavletic SZ. Impact of chronic graft-versus-host disease on late relapse and survival on 7,489 patients after myeloablative allogeneic hematopoietic cell transplantation for leukemia. Clinical Cancer Research. 2015 May 1; 21(9):2020-2028. doi:10.1158/1078-0432.CCR-14-0586. Epub 2014 Oct 27. PMC4411210. ncbi.nlm.nih.gov/pubmed/25348512






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7.3 Current Studies

GV16-01a Title: Graft-versus-host disease-free relapse-free survival in alternative donor

hematopoietic cell transplantation PIs: Rohtesh S. Mehta (M.D. Anderson Cancer Center) Shernan Holtan (University of Minnesota Blood and Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) GV17-01 Title: Investigating antibiotic exposure and risk of acute graft versus host disease

in children undergoing hematopoietic stem cell transplantation for acute leukemia

PIs: Caitlin Elgarten (Children's Hospital of Philadelphia) Brian Fisher (Children's Hospital of Philadelphia) Richard Aplenc (Children's Hospital of Philadelphia) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) GV17-02 Title: Risk factors of acute and chronic GVHD in T-replete HLA haploidnetical HCT

using post-transplantation cyclophosphamide PIs: Annie Im (University of Pittsburgh Medical Center) Betty Hamilton (Cleveland Clinic Foundation) Armin Rashidi (University of Minnesota) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) GV17-03 Title: Alterations in the characteristics and outcomes of acute and chronic GVHD

following post-transplant high dose cytoxan prophylaxis for haploidentical transplantation and in patients over 60 at high risk for GVHD

PIs: Rima Saliba (M.D. Anderson Cancer Center) Stefan Ciurea (M.D. Anderson Cancer Center) Jeff Schriber (Cancer Transplant Institute at Virginia G. Piper Cancer Center) Status: Analysis (as of July 1, 2019)



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GV18-01 Title: Comparison of late effects among allogeneic hematopoietic cell

transplantation survivors with and without chronic graft-versus-host disease

PIs: Catherine Lee (Utah Blood and Marrow Transplant Program) Daniel Couriel (Utah Blood and Marrow Transplant Program) Status: Draft Protocol Received (as of July 1, 2019)

Analysis (expected by June 30, 2020) GV18-02 Title: Comparion of antibacterial prophylaxis strategies and outcomes in

allogeneic stem cell transplantation patients with acute graft vs host disease PI: Whitney Wallis (Monroe Carrel Jr. Children's Hospital) Status: Protocol Development (as of July 1, 2019)

Analysis (expected by June 30, 2020) GV18-03 Title: Impact of chronic graft-versus-host disease on non-relapse mortality and

disease relapse in transplant recipients PIs: Vijaya Bhatt (Nebraska Medicine) Stephanie Lee (Fred Hutchinson Cancer Research Center) Status: Draft Protocol Received (as of July 1, 2019)

Analysis (expected by June 30, 2020) GV18-04 Title: Development of a risk score to predict the incidence of acute GVHD after

allogeneic hematopoietic cell transplantation PI: Guru Subramanian Guru Murthy (Medical College of Wisconsin) Status: Data File Preparation (as of July 1, 2019)


GV19-01 Title: Exploring the link between donor-engrafted clonal hematopoiesis and

adverse outcomes in allogeneic HCT recipients PIs: Nancy Gillis Johnson (H. Lee Moffitt Cancer Center and Research Institute) Eric Padron (H. Lee Moffitt Cancer Center and Research Institute) Aleksandr Lazaryan (H. Lee Moffitt Cancer Center and Research Institute) Status: Protocol Development (as of July 1, 2019)

Sanple Typing (expected by June 30, 2020)

2019 CIBMTR Scientific Working Committee Research Portfolio 8.0 HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE

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8.0 HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE

8.1 Leadership

Chair: Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital Email: [email protected] Chair: William Wood, MD, MPH, University of North Carolina Hospitals Email: [email protected] Chair: Shahrukh Hashmi, MD, MPH, King Faisal Specialist Hospital and Research

Center Email: [email protected]

Scientific Director: Wael Saber, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Naya He, MPH, CIBMTR Milwaukee Email: [email protected]


2019

Paulson K, Brazauskas R, Khera N, He N, Majhail N, Akpek G, Aljurf M, Buchbinder D, Burns L, Beattie S, Freytes C, Garcia A, Gajewski J, Hahn T, Knight J, LeMaistre C, Lazarus H, Szwajcer D, Seftel M, Wirk B, Wood W, Saber W. Inferior access to allogeneic transplant in disadvantaged populations: A CIBMTR analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.06.012. Epub 2019 Jun 19. ncbi.nlm.nih.gov/pubmed/31228584

2018

Elgarten CW, Arnold SD, Li Y, Huang Y-V, Riches ML, Gerber JS, Aplenc R, Saber W, Fisher BT. Hospital-level variability in broad-spectrum antibiotic use for children with acute leukemia undergoing hematopoietic cell transplantation. Infection Control and Hospital Epidemiology. doi:10.1017/ice.2018.96. Epub 2018 May 8. ncbi.nlm.nih.gov/pubmed/29734957









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Wood WA, Brazauskas R, Hu ZH, Abdel-Azim H, Ahmed IA, Aljurf M, Badawy S, Beitinjaneh A, George B, Buchbinder D, Cerny J, Dedeken L, Diaz MA, Freytes CO, Ganguly S, Gergis U, Almaguer DG, Gupta A, Hale G, Hashmi S, Inamoto Y, Kamble RT, Adekola K, Kindwall-Keller T, Knight J, Kumar L, Kuwatsuka Y, Law J, Lazarus HM, LeMaistre C, Olsson RF, Pulsipher MA, Savani BN, Schultz KR, Saad A, Seftel M, Seo S, Shea TC, Steinberg A, Sullivan K, Szwajcer D, Wirk B, Yared J, Yong A, Dalal J, Hahn T, Khera N, Bonfim C, Atsuta Y, Saber W. Country-level macroeconomic indicators predict early post-allogeneic hematopoietic cell transplantation survival in acute lymphoblastic leukemia: A CIBMTR analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.03.016. Epub 2018 Mar 19. ncbi.nlm.nih.gov/pubmed/29567340

2017

Arnold SD, Brazauskas R, He N, Li Y, Aplenc R, Jin Z, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Majhail NS, Diaz MA, Freytes CO, Wood WA, Savani BM, Kamble RT, Parsons S, Ahmed I, Sullivan K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel-Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P. Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases. Haematologica. 2017 Nov 1; 102(11):1823-1832. doi:10.3324/haematol.2017.169581. Epub 2017 Aug 17. PMC5664386. ncbi.nlm.nih.gov/ pubmed/28818869

El-Jawahri A, Chen Y-B, Brazauskas R, He N, Lee SJ, Knight JM, Majhail N, Buchbinder D, Schears RM, Wirk BM, Wood WA, Ahmed I, Aljurf M, Szer J, Beattie SM, Battiwalla M, Dandoy C, Diaz M-A, D'Souza A, Freytes CO, Gajewski J, Gergis U, Hashmi SK, Jakubowski A, Kamble RT, Kindwall-Keller T, Lazarus HM, Malone AK, Marks DI, Meehan K, Savani BN, Olsson RF, Rizzieri D, Steinberg A, Speckhart D, Szwajcer D, Schoemans H, Seo S, Ustun C, Atsuta Y, Dalal J, Sales-Bonfim C, Khera N, Hahn T, Saber W. Impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation. Cancer. 2017 May 15; 123(10): 1828-1838. doi:10.1002/cncr.30546. Epub 2017 Jan 19. PMC5419891. ncbi.nlm.nih.gov /pubmed/28102896

2016

Wood WA, McGinn MK, Wilson D, Deal AM, Khera N, Shea TC, Devine SM, Appelbaum FR, Horowitz MM, Lee SJ. Practice patterns and preferences among hematopoietic cell transplantation clinicians. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Nov 1; 22(11):2092-2099. doi:10.1016/j.bbmt.2016.07.014. Epub 2016 Jul 29. PMC5067220. ncbi.nlm.nih.gov/ pubmed/27481447









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Denzen EM, Thao V, Hahn T, Lee SJ, McCarthy PL, Rizzo JD, Ammi M, Drexler R, Flesch S, James H, Omondi N, Murphy E, Pederson K, Majhail NS. Financial impact of allogeneic hematopoietic cell transplantation on patients and families over 2 years: Results from a multicenter pilot study. Bone Marrow Transplantation. doi:10.1038/bmt.2016.103. Epub 2016 Apr 18. ncbi.nlm.nih.gov/pubmed/27088381

Gale RP, Seber A, Bonfim C, Pasquini M. Haematopoietic cell transplants in Latin America. Bone Marrow Transplantation. 2016 Jul 1; 51(7):898-905. doi:10.1038/bmt.2016.35. Epub 2016 Mar 21. PMC4935585. ncbi.nlm.nih.gov/pubmed/26999468

Niederwieser D, Baldomero H, Szer J, Gratwohl M, Aljurf M, Atsuta Y, Bouzas LF, Confer D, Greinix H, Horowitz M, Iida M, Lipton J, Mohty M, Novitzky N, Nunez J, Passweg J, Pasquini MC, Kodera Y, Apperley J, Seber A, Gratwohl A. Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplantation. 2016 Jun 1; 51(6):778-785. doi:10.1038/bmt.2016.18. Epub 2016 Feb 22. PMC4889523. ncbi.nlm.nih.gov /pubmed/26901703

Kumar R, Kimura F, Ahn KW, Hu Z-H, Kuwatsuka Y, Klein JP, Pasquini M, Miyamura K, Kato K, Yoshimi A, Inamoto Y, Ichinohe T, Wood WA Jr, Wirk B, Seftel M, Rowlings P, Marks DI, Schultz KR, Gupta V, Dedeken L, George B, Cahn J-Y, Szer J, Lee JW, Ho AY, Fasth A, Hahn T, Khera N, Dalal J, Bonfim C, Aljurf M, Atsuta Y, Saber W. Comparing outcomes with bone marrow or peripheral blood stem cells as graft source for matched sibling transplants in severe aplastic anemia across different economic regions. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 May 1; 22(5):932-940. doi:10.1016/j.bbmt.2016.01.012. Epub 2016 Jan 18. PMC4826285. ncbi.nlm.nih.gov/pubmed/26797402

Yoshimi A, Baldomero H, Horowitz M, Szer J, Niederwieser D, Gratwohl A, Kodera Y. Global use of peripheral blood vs bone marrow as source of stem cells for allogeneic transplantation in patients with bone marrow failure. JAMA: The Journal of the American Medical Association. 2016 Jan 12; 315(2):198-200. doi:10.1001/jama.2015.13706. Epub 2016 Jan 12. ncbi.nlm.nih.gov/pubmed/26757470

Kanda J, Brazauskas R, Hu ZH, Kuwatsuka Y, Nagafuji K, Kanamori H, Kanda Y, Miyamura K, Murata M, Fukuda T, Sakamaki H, Kimura F, Seo S, Aljurf M, Yoshimi A, Milone G, Wood WA, Ustun C, Hashmi S, Pasquini M, Bonfim C, Dalal J, Hahn T, Atsuta Y, Saber W. GVHD after HLA-matched sibling BMT or PBSCT: Comparison of North American Caucasian and Japanese populations. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):744-751. doi:10.1016/j.bbmt.2015.12. 027. Epub 2016 Jan 4. PMC4801761. ncbi.nlm.nih.gov/pubmed/26762681

2015

Knight JM, Rizzo JD, Logan BR, Wang T, Arevalo JMG, Ma J, Cole SW. Low socioeconomic status, adverse gene expression profiles, and clinical outcomes in hematopoietic stem cell transplant recipients. Clinical Cancer Research. 2016 Jan 1; 22(1):69-78. doi:10.1158/1078-0432.CCR-15-1344. Epub 2015 Aug 18. PMC4703514. ncbi.nlm.nih.gov/pubmed/26286914










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Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ. Comparison of characteristics and outcomes of trial participants and nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 trial. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1815-1822. doi:10.1016/j.bbmt.2015.06.004. Epub 2015 Jun 11. PMC4568172. ncbi.nlm.nih.gov/pubmed/26071866

2014

Ballen KK, Joffe S, Brazauskas R, Wang Z, Aljurf MD, Akpek G, Dandoy C, Frangoul HA, Freytes CO, Khera N, Lazarus HM, LeMaistre CF, Mehta P, Parsons SK, Szwajcer D, Ustun C, Wood WA, Majhail NS. Hospital length of stay in the first 100 days after allogeneic hematopoietic cell transplantation for acute leukemia in remission: Comparison among alternative graft sources. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1819-1827. doi:10.1016/j.bbmt. 2014.07.021. Epub 2014 Jul 23. PMC4194253. ncbi.nlm.nih.gov/pubmed/25064747

8.3 Current Studies

HS14-01 Title: Investigating clinical outcomes and inpatient health care resource utilization of

hematopoietic cell transplantation for children with acute leukemia PIs: Staci Arnold (Emory University Hospital) Richard Aplenc (Children's Hospital of Philadelphia) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with Pediatric Health Information System (PHIS) HS15-01 Title: Who is lost to follow-up in the Center for International Blood and Marrow

Transplant Research (CIBMTR) registry? PI: David Buchbinder (Children's Hospital of Orange County) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) HS15-02 Title: Impact of socioeconomic status on pediatric stem cell transplant outcomes PI: Kira Bona (Dana Farber Cancer Institute) Status: Manuscript Preparation (as of July 1, 2019)





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HS16-01 Title: Trends in utilization and outcomes of autologous and allogeneic hematopoietic

cell transplantation in racial and ethnic minorities PIs: Nandita Khera (Mayo Clinic Arizona and Phoenix Children's Hospital) Wael Saber (Medical College of Wisconsin) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) HS16-02 Title: The impact of marital status on hematopoietic stem cell transplant recipient

outcomes: A surrogate for consistent caregiver PI: Sara Margaret Beattie (Tom Baker Cancer Centre) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) HS16-03 Title: Relationship of race / ethnicity and survival after single and double umbilical

cord blood transplantation PI: Karen Ballen (University of Virginia Health System) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) HS17-01 Title: Association of community health status and center survival for allogeneic

hematopoietic cell transplantation PIs: Sanghee Hong (Cleveland Clinic Foundation) Navneet Singh Majhail (Cleveland Clinic Foundation) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with PHIS HS18-01 Title: International collaborative study to compare the prognosis for acute leukemia

patients transplanted with intensified myeloablative regimens PIs: Yasuyuki Arai (Kyoto University) Yoshiko Atsuta (Nagoya University Graduate School of Medicine) Shingo Yano (Jikei University School of Medicine) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with the Japan Society for Hematopoietic Cell Transplantation


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HS18-02 Title: Racial differences in long term survivor outcomes after allogeneic transplants PIs: Branson Blue (Saint Louis University) Navneet Majhail (Cleveland Clinic Foundation) Status: Protocol Development (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) HS18-03 Title: Racial / ethnic disparitites in receipt of hematopoietic celll transplantation and

subsequesnt resource utilization in children with acute leukemia PIs: Lena Winestone (Children's Hospital of Philadelphia) Richard Aplenc (Children's Hospital of Philadelphia) Kelly Getz (Perelman School of Medicine, University of Pennsylvania) Status: Protocol Development (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with PHIS HS19-01 Title: Factors associated with clinical trial participation among HSCT patients: a

CIBMTR Analysis PIs: Tamryn Gray (Dana Farber Cancer Institute) Areej El-Jawahri (Massachusetts General Hospital) Status: Protocol Development (as of July 1, 2019)

Protocol Development (expected by June 30, 2020) HS19-02 Title: Comparing outcomes of myeloablative T-replete haploidentical transplantation

with PT-CY protocol and ATG+G-CSF Protocol in patients with cytogenetic intermediate / high risk acute myeloid leukemia in first complete remission

PI: Xiao-Jun Huang (Peking Univeristy Institute of Hematology) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Peking University HS19-03 Title: Haploidentical stem cell transplantation for malignant and non-malignant

hematological diseases in patients without sibling donor: A multicenter prospective longitudinal study of the Brazilian bone marrow transplantation study group

PIs: Nelson Hamerschlak (M.D. Anderson Cancer Center) Mariana Kerbauy (Hospital Israelita Albert Einstein) Andreza Ribeiro (Hospital Israelita Albert Einstein) Status: Data Collection (as of July 1, 2019)

Data Collection (expected by June 30, 2020) * Collaborative study with the Brazilian Transplant Group


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HS19-04 Title: Outcomes after allogeneic stem cell transplants performed in Brazil from HLA-

matched siblings, unrelated and mismatched related donors: Retrospective study on behalf of the Brazilian Bone Marrow Transplantation Society (SBTMO), GEDECo (Brazil-Seattle Transplant-related complications Consortium), Hospital Israelita Albert Einstein (AmigoH), Associação da Medula Óssea do Estado de São Paulo (Ameo), Program Nacional de Apoio à Atenção Oncológica (Pronon), and CIBMTR

PIs: Adriana Seber (Hospital Samaritano) Nelson Hamerschlak (M.D. Anderson Cancer Center) Mary Flowers (Fred Hutchinson Cancer Research Center) Marcelo Pasquini (Medical College of Wisconsin) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with the Brazilian Transplant Group

2019 CIBMTR Scientific Working Committee Research Portfolio 9.0 IMMUNOBIOLOGY WORKING COMMITTEE

Page | 49

9.0 IMMUNOBIOLOGY WORKING COMMITTEE

9.1 Leadership

Chair: Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center Email: [email protected] Chair: Sophie Paczesny, MD, MPH, Indiana University Hospital / Riley Hospital

for Children Email: [email protected] Chair: Steven Marsh, BSc, PhD, ARCS, Anthony Nolan Research Institute Email: [email protected] Scientific Director: Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research

Center Email: [email protected] Scientific Director: Stephen Spellman, MBS, CIBMTR Minneapolis Email: [email protected] Statistical Director: Tao Wang, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Michelle Kuxhausen, MS, CIBMTR Minneapolis Email: [email protected]


2018

Bachanova V, Weisdorf DJ, Wang T, Marsh SGE, Cereb N, Haagenson MD, Spellman SR, Lee SJ, Guethlein LA, Parham P, Miller JS, Cooley S. Donor killer-cell immunoglobulin-like receptor (KIR) genotype does not improve graft-versus-leukemia responses in chronic lymphocytic leukemia (CLL) after unrelated donor transplant: A CIBMTR analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 May 1; 25(5):949-954. doi:10.1016/j.bbmt.2018.12.763. Epub 2018 Dec 27. PMC6511301. ncbi.nlm.nih.gov/pubmed/30594542

Askar M, Sayer D, Wang T, Haagenson M, Spellman SR, Lee SJ, Madbouly A, Fleischhauer K, Hsu KC, Verneris MR, Thomas D, Zhang A, Sobecks RM, Majhail NS. Analysis of single nucleotide polymorphisms in the gamma block of the major histocompatibility complex in association with clinical outcomes of hematopoietic cell transplantation: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Apr 1; 25(4):664-672. doi:10.1016/j.bbmt.2018.12.008. Epub 2018 Dec 8. PMC6453713. ncbi.nlm.nih.gov/pubmed/30537553











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Wang Y, Savage SA, Alsaggaf R, Aubert G, Dagnall CL, Spellman SR, Lee SJ, Hicks B, Jones K, Katki HA, Gadalla SM. Telomere length calibration from qPCR measurement: Limitations of current method. Cells. 2018 Oct 24; 7(11):e183. doi:10.3390/cells7110183. Epub 2018 Oct 24. PMC6262465. ncbi.nlm.nih.gov/pubmed/30352968

Pingel J, Wang T, Hagenlocher Y, Hernández-Frederick CJ, Nagler A, Haagenson MD, Fleischhauer K, Hsu KC, Verneris MR, Lee SJ, Mohty M, Polge E, Spellman SR, Schmidt AH, van Rood JJ. The effect of NIMA matching in adult unrelated mismatched hematopoietic stem cell transplantation - a joint study of the Acute Leukemia Working Party of the EBMT and the CIBMTR. Bone Marrow Transplantation. 2019 Jun 1; 54(6):849-857. doi:10.1038/s41409-018-0345-8. Epub 2018 Oct 2. PMC6445791. ncbi.nlm.nih.gov/pubmed/30279575

Woolfrey A, Wang T, Lee SJ, Haagenson MD, Chen G, Fleischhauer K, Horan J, Hsu K, Verneris M, Spellman SR, Fernandez-Vina M. Donor-specific anti-HLA antibodies in unrelated hematopoietic cell transplantation for non-malignant disorders. Bone Marrow Transplantation. 2019 Mar 1; 54(3):494-496. doi:10.1038/s41409-018-0334-y. Epub 2018 Sep 19. PMC6420380. ncbi.nlm.nih.gov/pubmed/30232414

Weisdorf D, Cooley S, Wang T, Trachtenberg E, Haagenson MD, Vierra-Green C, Spellman SR, Spahn A, Vogel J, Kobusingye H, Fehninger T, Woolfrey A, Devine S, Ross M, Waller EK, Sobecks R, Parham P, Guethlein LA, Marsh SGE, Miller J. KIR donor selection: Feasibility in identifying better donors. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jan 1; 25(1):e28-e32. doi:10.1016/j.bbmt.2018.08. 022. Epub 2018 Aug 24. PMC6310641. ncbi.nlm.nih.gov/pubmed/30149149

Arrieta-Bolaños E, Crivello P, Shaw BE, Ahn KW, Wang H-L, Verneris MR, Hsu KC, Pidala J, Lee SJ, Fleischhauer K, Spellman SR. In silico prediction of nonpermissive HLA-DPB1 mismatches in unrelated HCT by functional distance. Blood Advances. 2018 Jul 24; 2(14):1773-1783. doi:10.1182/bloodadvances.2018019620. Epub 2018 Jul 24. PMC6058232. ncbi.nlm.nih.gov/pubmed/30042143

Sarhan D, Brandt L, Felices M, Guldevall K, Lenvik T, Hinderlie P, Curtsinger J, Warlick E, Spellman SR, Blazar BR, Weisdorf DJ, Cooley S, Vallera DA, Önfelt B, Miller JS. 161533 TriKE stimulates NK-cell function to overcome myeloid-derived suppressor cells in MDS. Blood Advances. 2018 Jun 26; 2(12):1459-1469. doi:10.1182/bloodadvances.2017012369. PMC6020813. ncbi.nlm.nih.gov/pmc/articles/PMC6020813/

Petersdorf EW, Stevenson P, Malkki M, Strong RK, Spellman SR, Haagenson MD, Horowitz MM, Gooley T, Wang T. Patient HLA germline variation and transplant survivorship. Journal of Clinical Oncology. doi:10.1200/JCO.2017.77.6534. Epub 2018 Jun 14. ncbi.nlm.nih.gov/pubmed/ 29902106

Wang Y, Zhou W, Alter BP, Wang T, Spellman SR, Haagenson M, Yeager M, Lee SJ, Chanock SJ, Savage SA, Gadalla SM. Chromosomal aberrations and survival after unrelated donor hematopoietic stem cell transplant in patients with Fanconi anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.05.027. Epub 2018 Jun 4. ncbi.nlm.nih.gov/pubmed/ 29879518






http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6020813/






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Fleischhauer K, Hsu KC, Shaw BE. Prevention of relapse after allogeneic hematopoietic cell transplantation by donor and cell source selection. Bone Marrow Transplantation. doi:10.1038/s41409-018-0218-1. Epub 2018 May 24. ncbi.nlm.nih.gov/pubmed/29795435

Gadalla SM, Aubert G, Wang T, Haagenson M, Spellman SR, Wang L, Katki HA, Savage SA, Lee SJ. Donor telomere length and causes of death after unrelated hematopoietic cell transplant in patients with marrow failure. Blood. 2018 May 31; 131(21):2393-2398. doi:10.1182/blood-2017-10-812735. Epub 2018 Apr 9. PMC5969378. ncbi.nlm.nih.gov/pubmed/29632022

Zhu Q, Yan L, Liu Q, Zhang C, Wei L, Hu Q, Preus L, Clay-Gilmour AI, Onel K, Stram DO, Pooler L, Sheng X, Haiman CA, Zhu X, Spellman SR, Pasquini MC, McCarthy P, Liu S, Hahn T, Sucheston-Campbell LE. Exome chip analyses identify genes affecting mortality after HLA-matched unrelated donor blood and marrow transplantation. Blood. 2018 May 31; 131(22):2490-2499. doi:10.1182/blood-2017-11-817973. Epub 2018 Apr 2. PMC5981168. ncbi.nlm.nih.gov/ pubmed/29610366

Shaw BE, Logan BR, Spellman SR, Marsh SGE, Robinson J, Pidala J, Hurley C, Barker J, Maiers M, Dehn J, Wang H, Haagenson M, Porter D, Petersdorf EW, Woolfrey A, Horowitz MM, Verneris M, Hsu KC, Fleischhauer K, Lee SJ. Development of an unrelated donor selection score predictive of survival after HCT: Donor age matters most. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 May 1; 24(5):1049-1056. doi:10.1016/j.bbmt.2018.02.006. Epub 2018 Feb 14. PMC5953795. ncbi.nlm.nih.gov/pubmed/29454040

Buturovic L, Shelton J, Spellman SR, Wang T, Friedman L, Loftus D, Hesterberg L, Woodring T, Fleischhauer K, Hsu KC, Verneris MR, Haagenson M, Lee SJ. Evaluation of a machine learning-based prognostic model for unrelated hematopoietic cell transplantation donor selection. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jun 1; 24(6):1299-1306. doi:10.1016/j.bbmt.2018.01.038. Epub 2018 Feb 1. PMC5993610. ncbi.nlm.nih.gov/pubmed/29410341

Lee C, Haneuse S, Wang H-L, Rose S, Spellman SR, Verneris MR, Hsu KC, Fleischhauer K, Lee SJ, Abdi R. Prediction of acute graft-versus-host disease following hematopoietic cell transplantation. PLoS One. 13(1):e0190610. doi:10.1371/journal.pone.0190610. Epub 2018 Jan 18. PMC5773230. ncbi.nlm.nih.gov/pubmed/29346409

2017

Gadalla SM, Wang T, Loftus D, Friedman L, Dagnall C, Haagenson M, Spellman SR, Buturovic L, Blauwkamp M, Shelton J, Fleischhauer K, Hsu KC, Verneris MR, Krstajic D, Hicks B, Jones K, Lee SJ, Savage SA. No association between donor telomere length and outcomes after allogeneic unrelated hematopoietic cell transplant in patients with acute leukemia. Bone Marrow Transplantation. 2018 Apr 1; 53(4):383-391. doi:10.1038/s41409-017-0029-9. Epub 2017 Dec 21. PMC5898974. ncbi.nlm.nih.gov/pubmed/29269807










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William BM, Wang T, Haagenson MD, Fleischhauer K, Verneris M, Hsu KC, de Lima MJ, Fernandez-Viña M, Spellman SR, Lee SJ, Hill BT. Impact of HLA alleles on outcomes of allogeneic transplantation for B cell non-Hodgkin lymphomas: A Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Apr 1; 24(4):827-831. doi:10.1016/j.bbmt.2017.11.003. Epub 2017 Nov 16. PMC5902644. ncbi.nlm.nih.gov/pubmed/ 29155319

Clay-Gilmour AI, Hahn T, Preus LM, Onel K, Skol A, Hungate E, Zhu Q, Haiman CA, Stram DO, Pooler L, Sheng X, Yan L, Liu Q, Hu Q, Liu S, Battaglia S, Zhu X, Block AW, Suit SNJ, Karaesmen E, Rizvi A, Weisdorf DJ, Ambrosone CB, Tritchler D, Ellinghaus E, Ellinghaus D, Stanulla M, Clavel J, Orsi L, Spellman S, Pasquini MC, McCarthy PL, Sucheston-Campbell LE. Genetic association with B-cell acute lymphoblastic leukemia in allogeneic transplant patients differs by age and sex. Blood Advances. 2017 Sep 12; 1(20):1717-1728. doi:10.1182/bloodadvances.2017006023. Epub 2017 Sep 8. PMC5728332. ncbi.nlm.nih.gov/pubmed/29296818

Fleischhauer K, Shaw BE. HLA-DP in unrelated hematopoietic cell transplantation revisited: Challenges and opportunities. Blood. 2017 Aug 1; 130(9):1089-1096. doi:10.1182/blood-2017-03-742346. Epub 2017 Jun 30. ncbi.nlm.nih.gov/pubmed/28667011

Eapen M, Wang T, Veys PA, Boelens JJ, St, Martin A, Spellman S, Bonfim C, Brady C, Cant AJ, Dalle J-H, Davies SM, Freeman J, Hsu KC, Fleischhauer K, Kenzey C, Kurtzberg J, Michel G, Orchard PJ, Paviglianiti A, Rocha V, Veneris M, Volt F, Wynn R, Lee SJ, Horowitz MM, Gluckman E, Ruggeri A. Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: A retrospective analysis. The Lancet Haematology. 2017 Jul 1; 4(7):e325-e333. doi:10.1016/S2352-3026(17)30104-7. Epub 2017 Jun 13. PMC5699478. ncbi.nlm.nih.gov/pubmed/28623181

Fleischhauer K, Ahn KW, Wang HL, Zito L, Crivello P, Müller C, Verneris M, Shaw BE, Pidala J, Oudshorn M, Lee SJ, Spellman SR. Directionality of non-permissive HLA-DPB1 T-cell epitope group mismatches does not improve clinical risk stratification in 8/8 matched unrelated donor hematopoietic cell transplantation. Bone Marrow Transplantation. 2017 Sep 1; 52(9):1280-1287. doi:10.1038/bmt.2017.96. Epub 2017 Jun 5. PMC5968827. ncbi.nlm.nih.gov/ pubmed/28581467

Boudreau JE, Giglio F, Gooley TA, Stevenson PA, Le Luduec J-B, Shaffer BC, Rajalingam R, Hou L, Hurley CK, Noreen H, Reed EF, Yu N, Vierra-Green C, Haagenson M, Malkki M, Petersdorf EW, Spellman S, Hsu K. KIR3DL1/HLA-B subtypes govern acute myelogenous leukemia relapse after hematopoietic cell transplantation. Journal of Clinical Oncology. 2017 Jul 1; 35(20):2268-2278. doi:10.1200/JCO.2016.70.7059. Epub 2017 May 18. PMC5501362. ncbi.nlm.nih.gov/pubmed/28520526

Wang Y, Wang T, Dagnall C, Haagenson M, Spellman SR, Hicks B, Jones K, Lee SJ, Savage SA, Gadalla SM. Relative telomere length prior to hematopoietic cell transplantation (HCT) and outcome after unrelated donor HCT for acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jul 1; 23(7):1054-1058. doi:10.1016/j.bbmt.2017.03.032. Epub 2017 Apr 4. PMC5657243. ncbi.nlm.nih.gov/pubmed/28389255











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Madbouly A, Wang T, Haagenson M, Paunic V, Vierra-Green C, Fleischhauer K, Hsu KC, Verneris MR. Investigating the association of genetic admixture and donor/recipient genetic disparity with transplant outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jun 1; 23(6):1029-1037. doi:10.1016/j.bbmt.2017.02.019. Epub 2017 Mar 2. PMC5541944. ncbi.nlm.nih.gov/pubmed/ 28263917

Lindsley RC, Saber W, Mar BG, Redd R, Wang T, Haagenson MD, Grauman PV, Hu Z-H, Spellman SR, Lee SJ, Verneris MR, Hsu K, Fleischhauer K, Cutler C, Antin JH, Neuberg D, Ebert BL. Prognostic mutations in myelodysplastic syndrome after stem-cell transplantation. New England Journal of Medicine. 2017 Feb 9; 376(6):536-547. doi:10.1056/NEJMoa1611604. Epub 2017 Feb 9. PMC5438571. ncbi.nlm.nih.gov/pubmed/28177873

2016

Hou L, Vierra-Green C, Lazaro A, Brady C, Haagenson M, Spellman S, Hurley CK. Limited HLA sequence variation outside of antigen recognition domain exons of 360 10 of 10 matched unrelated hematopoietic stem cell transplant donor-recipient pairs. HLA. 2017 Jan 1; 89(1):39-46. doi:10.1111/tan.12942. Epub 2016 Dec 15. PMC5425813. ncbi.nlm.nih.gov/ pubmed/27976839

Askar M, Sobecks R, Wang T, Haagenson M, Majhail N, Madbouly A, Thomas D, Zhang A, Fleischhauer K, Hsu K, Verneris M, Lee SJ, Spellman SR, Fernández-Viña M. MHC class I chain-related gene A (MICA) donor-recipient mismatches and MICA-129 polymorphism in unrelated donor hematopoietic cell transplantations has no impact on outcomes in acute lymphoblastic leukemia, acute myeloid leukemia, or myelodysplastic syndrome: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Mar 1; 23(3):436-444. doi:10.1016/j.bbmt.2016.11.021. Epub 2016 Dec 14. PMC5370205. ncbi.nlm.nih.gov/pubmed/27987385

Hoff GA, Fischer JC, Hsu K, Cooley S, Miller JS, Wang T, Haagenson M, Spellman S, Lee SJ, Uhrberg M, Venstrom JM, Verneris MR. Recipient HLA-C haplotypes and microRNA 148a/b binding sites have no impact on allogeneic hematopoietic cell transplantation outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):153-160. doi:10.1016/j.bbmt.2016.09.028. Epub 2016 Oct 13. PMC5198579. ncbi.nlm.nih.gov/pubmed/27746218

Goyal RK, Lee SJ, Wang T, Trucco M, Haagenson M, Spellman SR, Verneris M, Ferrell RE. Novel HLA-DP region susceptibility loci associated with severe acute GvHD. Bone Marrow Transplantation. 2017 Jan 1; 52(1):95-100. doi:10.1038/bmt.2016.210. Epub 2016 Sep 5. PMC5215038. ncbi.nlm.nih.gov/pubmed/27595289

Kornblit B, Wang T, Lee SJ, Spellman SR, Zhu X, Fleischhauer K, Müller C, Verneris MR, Müller K, Johansen JS, Vindelov L, Garred P. YKL-40 in allogeneic hematopoietic cell transplantation after AML and myelodysplastic syndrome. Bone Marrow Transplantation. 2016 Dec 1; 51(12):1556-1560. doi:10.1038/bmt.2016.192. Epub 2016 Jul 18. PMC5143177. ncbi.nlm.nih.gov/pubmed/27427920











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Lazaryan A, Wang T, Spellman SR, Wang H-L, Pidala J, Nishihori T, Askar M, Olsson R, Oudshoorn M, Abdel-Azim H, Yong A, Gandhi M, Dandoy C, Savani B, Hale G, Page K, Bitan M, Reshef R, Drobyski W, Marsh SGE, Schultz K, Müller CR, Fernandez-Viña M, Verneris MR, Horowitz MM, Arora M, Weisdorf DJ, Lee SJ. Human leukocyte antigen supertype matching after myeloablative hematopoietic cell transplantation with 7/8 matched unrelated donor allografts: A report from the Center for International Blood and Marrow Transplant Research. Haematologica. doi:10.3324/haematol.2016.143271. Epub 2016 May 31. ncbi.nlm.nih.gov/ pubmed/27247320

Marino SR, Lee SM, Binkowski TA, Wang T, Haagenson M, Wang H-L, Maiers M, Spellman S, van Besien K, Lee SJ, Karrison T, Artz A. Identification of high-risk amino-acid substitutions in hematopoietic cell transplantation: A challenging task. Bone Marrow Transplantation. doi:10.1038/bmt.2016.142. Epub 2016 May 23. ncbi.nlm.nih.gov/pubmed/27214075

Bachanova V, Weisdorf DJ, Wang T, Marsh SGE, Trachtenberg E, Haagenson MD, Spellman SR, Ladner M, Guethlein LA, Parham P, Miller JS, Cooley SA. Donor KIR B genotype improves progression free survival of non-Hodgkin lymphoma patients receiving unrelated donor transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.05.016. Epub 2016 May 21. ncbi.nlm.nih.gov/pubmed/27220262

Arora M, Lee SJ, Spellman SR, Weisdorf DJ, Guan W, Haagenson M, Wang T, Horowitz MH, Verneris MR, Fleischhauer K, Hsu K, Thyagarajan B. A validation study failed to confirm association between genetic variants in the base excision repair pathway and TRM and relapse post HCT. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.04.020. Epub 2016 May 4. ncbi.nlm.nih.gov/pubmed/27155583

Rocha V, Ruggeri A, Spellman S, Wang T, Sobecks R, Locatelli F, Askar M, Michel G, Arcese W, Iori AP, Purtill D, Danby R, Sanz GF, Gluckman E, Eapen M. Killer cell immunoglobulin-like receptor ligand matching and outcomes after unrelated cord blood transplantation acute myeloid leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.04.007. Epub 2016 Apr 16. ncbi.nlm.nih.gov/pubmed/27090957

Gadalla SM, Khincha PP, Katki HA, Giri N, Wong JYY, Spellman S, Yanovski JA, Han JC, De Vivo, Alter BP, Savage SA. The limitations of qPCR telomere length measurement in diagnosing dyskeratosis congenita. Molecular Genetics & Genomic Medicine. 2016 Jul 1; 4(4):475-479. doi:10.1002/mgg3.220. Epub 2016 Mar 20. PMC4947866. ncbi.nlm.nih.gov/pubmed/27468421

Nakasone H, Sahaf B, Tian L, Wang T, Haagenson MD, Schoenrock K, Perloff S, Ryan CE, Wu F, Spellman SR, Lee SJ, Ritz J, Miklos DB. Presensitization to HY antigens in female donors prior to transplant is not associated with male recipient post-transplant HY antibody development nor with clinical outcomes. Haematologica. 101(1):e30-e33. doi:10.3324/ haematol.2015.134551. Epub 2016 Jan 1. PMC4697905. ncbi.nlm.nih.gov/pubmed/26494841










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2015

Jindra PT, Conway SE, Ricklefs SM, Porcella SF, Anzick SL, Haagenson M, Wang T, Spellman S, Milford E, Kraft P, McDermott DH, Abdi R. Analysis of a genetic polymorphism in the costimulatory molecule TNFSF4 with hematopoietic stem cell transplant outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jan 1; 22(1):27-36. doi:10.1016/j.bbmt.2015.08.037. Epub 2015 Sep 5. PMC4743880. ncbi.nlm.nih.gov/pubmed/26348892

Petersdorf EW, Malkki M, O'hUigin C, Carrington M, Gooley T, Haagenson MD, Horowitz MM, Spellman SR, Wang T, Stevenson P. High HLA-DP expression and graft-versus-host disease. New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. doi:10.1056/NEJMoa1500140. Epub 2015 Aug 13. PMC4560117. ncbi.nlm.nih.gov/pubmed/26267621

Verneris MR, Lee SJ, Ahn KW, Wang H-L, Battiwalla M, Inamoto Y, Fernandez-Vina MA, Gajewski J, Pidala J, Munker R, Aljurf M, Saber W, Spellman S, Koreth J. HLA mismatch is associated with worse outcomes after unrelated donor reduced-intensity conditioning hematopoietic cell transplantation: An analysis from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1783-1789. doi:10.1016/ j.bbmt.2015.05.028. Epub 2015 Jun 6. PMC4568127. ncbi.nlm.nih.gov/pubmed/26055300

Sobecks RM, Wang T, Askar M, Gallagher MM, Haagenson M, Spellman S, Fernandez-Vina M, Malmberg K-J, Müller C, Battiwalla M, Gajewski J, Verneris MR, Ringdén O, Marino S, Davies S, Dehn J, Bornhäuser M, Inamoto Y, Woolfrey A, Shaw P, Pollack M, Weisdorf D, Milller J, Hurley C, Lee SJ, Hsu K. Impact of KIR and HLA genotypes on outcomes after reduced-intensity conditioning hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1589-1596. doi:10.1016/j.bbmt.2015.05.002. Epub 2015 May 8. PMC4537837. ncbi.nlm.nih.gov/pubmed/25960307

Gadalla SM, Wang T, Haagenson M, Spellman SR, Lee SJ, Williams KM, Wong JY, De Vivo I, Savage SA. Association between donor leukocyte telomere length and survival after unrelated allogeneic hematopoietic cell transplantation for severe aplastic anemia. JAMA: The Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. doi:10.1001/jama.2015.7. Epub 2015 Feb 10. PMC4388056. ncbi.nlm.nih.gov/pubmed/25668263

Logan AC, Wang Z, Alimoghaddam K, Wong RM, Lai T, Negrin RS, Grumet C, Logan BR, Zhang M-J, Spellman SR, Lee SJ, Miklos DB. ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):746-754. doi:10.1016/j.bbmt.2014.12.036. Epub 2015 Jan 5. PMC4363312. ncbi.nlm.nih.gov/pubmed/25572032

2014

Kornblit B, Enevold C, Wang T, Spellman S, Haagenson M, Lee SJ, Müller K. Toll-like receptor polymorphisms in allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):259-265. doi:10.1016/j.bbmt.2014.09.016. Epub 2014 Nov 20. PMC4297590. ncbi.nlm.nih.gov/pubmed/25464115









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Venstrom JM, Pittari G, Gooley TA, Chewning JH, Spellman S, Haagenson M, Gallagher MM, Malkki M, Petersdorf E, Dupont B, Hsu KC. Donor activating KIR2DS1 in leukemia. New England Journal of Medicine. 2014 Nov 20; 371(21):2042. doi:10.1056/NEJMc1411443. Epub 2014 Nov 20. ncbi.nlm.nih.gov/pubmed/25409391

Broux B, Shamim Z, Wang T, Spellman S, Haagenson M, Stinissen P, Ryder LP, Müller K, Hellings N. The influence of interleukin-7 receptor α-chain haplotypes on outcome after allogeneic hematopoietic cell transplantation. International Journal of Immunogenetics. 2014 Dec 1; 41(6):521-527. doi:10.1111/iji.12158. Epub 2014 Oct 29. PMC4238034. ncbi.nlm.nih.gov/ pubmed/25352021

Petersdorf EW, Gooley TA, Malkki M, Bacigalupo AP, Cesbron A, Du Toit E, Ehninger G, Egeland T, Fischer GF, Gervais T, Haagenson MD, Horowitz MM, Hsu K, Jindra P, Madrigal A, Oudshoorn M, Ringdén O, Schroeder ML, Spellman SR, Tiercy JM, Velardi A, Witt CS, O’hUigin C, Apps R, Carrington M. HLA-C expression levels define permissible mismatches in hematopoietic cell transplantation. Blood. 2014 Dec 18; 124(26):3996-4003. doi:10.1182/blood-2014-09-599969. Epub 2014 Oct 16. PMC4271183. ncbi.nlm.nih.gov/pubmed/25323824

Pidala J, Lee SJ, Ahn KW, Spellman S, Wang H-L, Aljurf M, Askar M, Dehn J, Fernandez Viña M, Gratwohl A, Gupta V, Hanna R, Horowitz MM, Hurley CK, Inamoto Y, Kassim AA, Nishihori T, Mueller C, Oudshoorn M, Petersdorf EW, Prasad V, Robinson J, Saber W, Schultz KR, Shaw B, Storek J, Wood WA, Woolfrey AE, Anasetti C. Nonpermissive HLA-DPB1 mismatch increases mortality after myeloablative unrelated allogeneic hematopoietic cell transplantation. Blood. 2014 Oct 16; 124(16):2596-2606. doi:10.1182/blood-2014-05-576041. Epub 2014 Aug 26. PMC4199961. ncbi.nlm.nih.gov/pubmed/25161269

9.3 Current Studies

IB06-05 Title: Use of high-resolution HLA data from the NMDP for the International

Histocompatibility Working Group in HCT PI: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with the International Histocompatibility Working Group IB09-01 Title: Clinical importance of minor histocompatibility complex haplotypes in umbilical

cord blood transplantation PI: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with the International Histocompatibility Working Group







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IB09-03 Title: Clinical relevance of cytokine/immune response gene polymorphisms in

umbilical cord blood transplantation PI: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with the International Histocompatibility Working Group IB09-05 Title: Identification of functional single nucleotide polymorphisms in umbilical cord

blood transplantation PI: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with the International Histocompatibility Working Group IB09-06b Title: Genetic susceptibility to transplant-related mortality after unrelated donor stem

cell transplant PI: Theresa Hahn (Roswell Park Cancer Institute) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute IB09-06f Title: DISCOVeRY-BMT: Case-control study of acute myelogenous leukemia exome and

genome-wide association study PIs: Alyssa Clay-Gilmour (Roswell Park Cancer Institute) Lara Sucheston-Cambpell (Ohio State Medical Center, James Cancer Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State IB09-06j Title: DISCOVeRY-BMT: Additional analysis of major histocompatibility complex single

nucleotide polymorphisms PIs: Steve Spellman (CIBMTR - Minneapolis) Lara Sucheston-Cambpell (Ohio State Medical Center, James Cancer Center) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State


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IB09-06m Title: DISCOVeRY-BMT: Analysis of X chromosome single nucleotide polymorphisms PIs: Steve Spellman (CIBMTR - Minneapolis) Lara Sucheston-Cambpell (Ohio State Medical Center, James Cancer Center) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State IB09-06n Title: DISCOVeRY-BMT: Compare unrelated donor to Welcome Trust Case Control

Consortium controls PIs: Kenan Onel (Comer Children's Hospital / University of Chicago Medicine) Alyssa Clay-Gilmour (Roswell Park Cancer Institute) Ezgi Karaesmen (Ohio State Medical Center, James Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State IB09-06o Title: DISCOVeRY-BMT: Genetics and Epidemiology of Myeloid Malignancies candidate

gene paper PIs: Lara Sucheston-Cambell (Ohio State Medical Center, James Cancer Center) Ezgi Karaesmen (Ohio State Medical Center, James Cancer Center) Alyssa Clay-Gilmour (Roswell Park Cancer Institute) Theresa Hahn (Roswell Park Cancer Institute) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State IB09-06p Title: DISCOVeRY-BMT: Genetics and Epidemiology of Myeloid Malignancies genome-

wide association study PIs: Alyssa Clay-Gilmour (Roswell Park Cancer Institute) Kenan Onel (Comer Children's Hospital / University of Chicago Medicine) Theresa Hahn (Roswell Park Cancer Institute) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State IB09-07 Title: Clinical significance of genome-wide variation in unrelated HCT PI: Effie Petersdorf (Fred Hutchinson Cancer Research Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with International Histocompatibility Working Group


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IB10-01f Title: Epigenetic clock: Can this guide donor selection in HCT PIs: Shahinaz Gadalla (National Cancer Institute) Sharon Savage (National Cancer Institute) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with the National Cancer Institute IB14-03a Title: The prognostic impact of levels of CXC chemokine ligands on post hematopoietic

cell transplantation outcomes in patients with myelodysplastic syndromes PIs: Wael Saber (Medical College of Wisconsin) Binod Dhakal (Medical College of Wisconsin) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) IB14-03c Title: Impact of telomere length and telomerase gene mutations on allogeneic stem cell

transplantation outcomes in myelodysplastic syndrome PIs: R. Coleman Lindsley (Dana Farber Cancer Institute at Brigham and Women's Hospital) Wael Saber (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) IB14-04 Title: Assessing the similarity of the T cell receptor repertoire in allogeneic

hematopoietic stem cell recipients with the same single human leukocyte mismatches

PI: Everett Meyer (Stanford Health Care) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) IB14-05 Title: mtDNA haplotypes and unrelated donor transplant outcomes PIs: Michael Verneris (University of Colorado - Children's Hospital) Logan Spector (University of Minnesota) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with University of Minnesota


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IB14-07 Title: Indirectly recognizable HLA epitopes (PIRCHES): A retrospective validation study

on the role of indirect recognition of mismatched HLA in hematopoietic stem cell transplantation outcome

PI: Eric Spierings (University Medical Center Utrecht) Status: Manuscript Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with International Histocompatibility Working Group IB15-03 Title: Killer immunoglobulin receptor (KIR) gene content and pediatric acute leukemia

transplant outcomes PIs: Michael R. Verneris (University of Colorado - Children's Hospital) Jeffrey Miller (University of Minnesota Blood and Marrow Transplant Program) Sarah Cooley (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with University of Minnesota IB15-04 Title: Clinical outcomes among hematopoietic stem cell transplant recipients as a

function of socioeconomic status and related transcriptome differences PIs: Jennifer Knight (Medical College of Wisconsin) J. Douglas Rizzo (Medical College of Wisconsin) Steve Cole (UCLA School of Medicine) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with the Medical College of Wisconsin IB16-01 Title: The role of HLA-E compatibility in the prognosis of acute leukemia patients

undergoing 10/10 HLA matched unrelated HSCT PIs: Chrysanthi Tsamadou (IKT Ulm) Daniel Furst (IKT Ulm) Joannis Mytilineos (IKT Ulm) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) IB16-02 Title: Use of HLA structure and function parameters to understand the relationship

between HLA disparity and transplant outcomes PI: Lee Ann Baxter Lowe (Children's Hospital of Los Angeles) Status: Manuscript Preparation (as of July 1, 2019)



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IB16-03 Title: Role of recipient and donor genetic polymorphisms in interferon lambda 4

(INFL4) on outcomes after unrelated allogeneic cell transplant PIs: Shahinaz M. Gadalla (National Cancer Institute) Ludmila Prokunina-Olsson (National Cancer Institute) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with the National Cancer Institute IB17-02 Title: Donor-recipient NK cell determinants associated with survival in JMML after

hematopoietic stem cell transplantation PIs: Dean Lee (Nationwide Children's Hospital) Hemalatha Rangarahan (Nationwide Children's Hospital) Status: Sample Typing (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) IB17-03 Title: Identification of genomic markers of post hematopoietic cell transplantation

(HCT) outcomes in patients with myelofibrosis: A pilot study PIs: Wael Saber (Medical College of Wisconsin) Shahinaz Gadalla (National Cancer Institute) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with the National Cancer Institute IB17-04 Title: Epigenetic profiling of unrelated donor-recipient pairs to improve donor

selection during HCT transplants PIs: Stephan Beck (University College London) Karl Peggs (University College London) Vardhman Rakyan (Barts and The London School of Medicine, Blizard Institute) Amy Webster (University College London) Status: Analysis (as of July 1, 2019)

Analysis (expected by June 30, 2020) IB18-01 Title: Effect of HLA phenotypes on long term GVHD risk PIs: Charlotte Story (University of North Carolina School of Medicine) Marcie Riches (University of North Carolina Hospitals) Paul Armistead (University of North Carolina Hospitals) Status: Data File Preparation (as of July 1, 2019)



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IB18-02 Title: The impact of HLA class I risk alleles associated with AA immune pathogenesis on

allogeneic transplant outcomes in patients with severe acquired aplastic anemia PIs: Daria Babushok (Children's Hospital of Philadelphia) Timothy Olson (Children's Hospital of Philadelphia) Status: Sample Typing (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) IB18-03 Title: The effect of HLA Class I heterozygosity and HLA supertypes on outcomes

following allogeneic hematopoietic cell transplant for myeloid and lymphoid malignancies

PIs: Christine Camacho-Bydume (Memorial Sloan Kettering Cancer Center) Katharine Hsu (Memorial Sloan Kettering Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) IB18-04 Title: Evaluation of the impact of donor KIR genotype on outcome after unrelated donor

transplantation in patients with myelodysplastic syndromes or secondary acute myeloid leukemia

PIs: Johannes Schetelig (Universitaetsklinikum Carl Gustav Carus) Nicolas Kröger (Universitaetsklinikum Hamburg, Eppendorf) Marie Robin (Hopital Saint Louis) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Deutsche Knochenmarkspenderdatei GmbH (DKMS, German Bone

Marrow Donor Center) IB18-05 Title: Imputation of KIR in genome-wide association study and the association of KIR-

HLA with outcomes following alloHCT In AML and MDS PIs: Christine Camacho-Bydume (Memorial Sloan Kettering Cancer Center) Lara Sucheston-Campbell (Ohio State Medical Center, James Cancer Center) Stephen Leslie (University of Melbourne) Katharine Hsu (Memorial Sloan Kettering Cancer Center) Status: Analysis (as of July 1, 2019)

Analysis (expected by June 30, 2020) * Collaborative study with International Histocompatibility Working Group


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IB18-06 Title: Clonal mosaicism and HCT outcomes in patients with acute leukemia and

myelodysplastic syndromes PIs: Shahinaz Gadalla (National Cancer Institute) Theresa Hahn (Roswell Park Cancer Institute) Lara Sucheston-Campbell (Ohio State Medical Center, James Cancer Center) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with the National Cancer Institute IB18-07 Title: Donor and recipient genomic associations with acute GVHD PI: Vahid Afshar-Khargan (M.D. Anderson Cancer Center) Status: Sample Typing (as of July 1, 2019)

Analysis (expected by June 30, 2020) * Collaborative study with the National Cancer Institute IB19-01 Title: The impact of ultra-high resolution HLA matching on the outcome of unrelated

donor hematopoietic cell transplantation PIs: Neema Mayor (Anthony Nolan Research Institute) Stephen Spellman (CIBMTR - Minneapolis) Steven Marsh (Anthony Nolan Research Institute) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) IB19-02 Title: Effect of class II HLA mismatching on the outcome of HLA-haploidentical

hematopoietic cell transplantation with high dose, post-transplantation cyclophosphamide: a combined CIBMTR/EBMT analysis

PIs: Shannon McCurdy (Hospital of the Unviersity of Pennsylvania) Scott Solomon (The Blood and Marrow Transplant Program at Northside Hospital) Yvette Kasamon (Johns Hopkins Hospital) Asad Bashey (The Blood and Marrow Transplant Program at Northside Hospital) Ephraim Fuchs (The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins) Status: Draft Protocol Received (as of July 1, 2019)



Page | 64

IB19-03 Title: Impact of the direction of NK cell alloreactivity predicted by KIR ligand mismatch

on engraftment in umbilical cord blood and haploidentical stem cell transplantation

PIs: Folashade Otegbeye (Abramson Cancer Center University of Pennsylvania Medical Center)

Marcelo Fernandez-Vina (Stanford Health Care) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) IB19-04 Title: Impact of donor HLA on transplant outcomes in NPM1 mutated AML PIs: Rupa Narayan (Massachusetts General Hospital) Everett Meyer (Stanford Health Care) Yi-Bin Chen (Massachusetts General Hospital) Status: Protocol Pending (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) IB19-05 Title: Impact of donor signal-regulatory protein alpha polymorphism on outcomes of

allogenetic hematopoietic stem cell transplantation PIs: Jayne Danska (The Hospital for Sick Children) Fadi Lakkis (University of Pittsburgh) Status: Draft Protocol Received (as of July 1, 2019)

Sample Typing (expected by June 30, 2020) * Collaborative study with The Hospital for Sick Children R02-40/R03-63d Title: Acquisition of natural killer cell receptors in recipients of unrelated transplant PI: Jeff Miller (University of Minnesota Blood and Marrow Transplant Program) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with University of Minnesota R04-74d Title: Functional significance of killer cell immunoglobulin-like receptor genes in HLA-

matched and mismatched unrelated HCT PI: Katharine Hsu (Memorial Sloan Kettering Cancer Center) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with International Histocompatibility Working Group


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RT09-04 / IB09-06i Title: DISCOVeRY-BMT: Recip, donor genome-wide association study interaction with

conditioning intensity (myeloablative / reduced intensity conditioning), total body irradiation, disease status

PIs: Ezgi Karaesmen (Ohio State Medical Center, James Cancer Center) Lara Sucheston-Cambpell (Ohio State Medical Center, James Cancer Center) Theresa Hahn (Roswell Park Cancer Institute) Status: Analysis (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Roswell Park Cancer Institute and Ohio State

2019 CIBMTR Scientific Working Committee Research Portfolio 10.0 INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE

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10.0 INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE

10.1 Leadership

Chair: Krishna Komanduri, MD, University of Miami Email: [email protected] Chair: Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center Email: [email protected] Chair: Roy Chemaly, MD, MPH, MD Anderson Cancer Center Email: [email protected] Scientific Director: Marcie Riches, MD, MS, University of North Carolina Hospitals Email: [email protected] Statistical Director: Soyoung Kim, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Naya He , MPH, CIBMTR Milwaukee Email: [email protected]


2019

Abidi MZ, Hari P, Chen M, Kim S, Battiwala M, Dahi PB, Diaz MA, Gale RP, Ganguly S, Gergis U, Green J, Hildebrandt G, Hill JA, Komanduri K, Lazarus H, Marks D, Nishihori T, Olsson R, Seo S, Ustun C, Yared J, Yin D, Wingard J, Wirk BM, Auletta J, Lindemans C, Riches M. Virus detection in the cerebrospinal fluid of hematopoietic stem cell transplant recipients is associated with poor patient outcomes: A CIBMTR contemporary longitudinal study. Bone Marrow Transplantation. doi:10.1038/s41409-019-0457-9. Epub 2019 Jan 29. ncbi.nlm.nih.gov/ pubmed/30696997

Papanicolaou GA, Ustun C, Young JH, Chen M, Kim S, Ahn KW, Komanduri K, Lindemans CA, Auletta JJ, Riches ML. Bloodstream infection (BSI) due to vancomycin-resistant enterococcus (VRE) is associated with increased mortality after hematopoietic cell transplantation for acute leukemia and myelodysplastic syndrome: A multicenter, retrospective cohort study. Clinical Infectious Diseases. doi:10.1093/cid/ciz031. Epub 2019 Jan 14. ncbi.nlm.nih.gov/pubmed/30649224











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2018

Ustun C, Young J-H, Papanicolaou GA, Kim S, Ahn KW, Chen M, Abdel-Azim H, Aljurf M, Beitinjaneh A, Brown V, Cerny J, Chhabra S, Kharfan-Dabaja MA, Dahi PB, Daly A, Dandoy CE, Dvorak CC, Freytes CO, Hashmi S, Lazarus H, Ljungman P, Nishihori T, Page K, Pingali SRK, Saad A, Savani BN, Weisdorf D, Williams K, Wirk B, Auletta JJ, Lindemans CA, Komanduri K, Riches M. Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. doi:10.1038/s41409-018-0401-4. Epub 2018 Dec 13. PMC6565512. ncbi.nlm.nih.gov/pubmed/30546070

2016

Maziarz RT, Brazauskas R, Chen M, McLeod AA, Martino R, Wingard JR, Aljurf M, Battiwalla M, Dvorak CC, Geroge B, Guinan EC, Hale GA, Lazarus HM, Lee J-W, Liesveld JL, Ramanathan M, Reddy V, Savani BN, Smith FO, Strasfeld L, Taplitz RA, Ustun C, Boeckh MJ, Gea-Banacloche J, Lindemans CA, Auletta JJ, Riches ML. Pre-existing invasive fungal infection is not a contraindication for allogeneic HSCT for patients with hematologic malignancies: A CIBMTR study. Bone Marrow Transplantation. 2017 Feb 1; 52(2):270-278. doi:10.1038/ bmt.2016.259. Epub 2016 Dec 19. PMC5358320. ncbi.nlm.nih.gov/pubmed/27991895

Ballen K, Ahn KW, Chen M, Abdel-Azim H, Ahmed I, Aljurf M, Antin J, Bhatt AS, Boeckh M, Chen G, Dandoy C, George B, Laughlin M, Lazarus HM, MacMillan ML, Margolis DA, Marks DI, Norkin M, Rosenthal J, Saad A, Savani B, Schouten HC, Storek J, Szabolcs P, Ustun C, Verneris MR, Waller EK, Weisdorf DJ, Williams KM, Wingard JR, Wirk B, Wolfs T, Young JH, Auletta J, Komanduri KV, Lindemans C, Riches ML. Infection rates among acute leukemia patients receiving alternative donor hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.06.012. Epub 2016 Jun 22. ncbi.nlm.nih.gov/pubmed/27343716

Ramanathan M, Teira P, Battiwalla M, Barrett J, Ahn KW, Chen M, Green J, Laughlin M, Lazarus HM, Marks D, Saad A, Seftel M, Saber W, Savani BN, Waller E, Wingard J, Auletta JJ, Lindemans CA, Boeckh M, Riches ML. Impact of early CMV reactivation in cord blood stem cell recipients in the current era. Bone Marrow Transplantation. doi:10.1038/bmt.2016.89. Epub 2016 Apr 4. ncbi.nlm.nih.gov/pubmed/27042847

Teira P, Battiwalla M, Ramanathan M, Barrett AJ, Ahn KW, Chen M, Green JS, Saad A, Antin JH, Savani BN, Lazarus HM, Seftel M, Saber W, Marks D, Aljurf M, Norkin M, Wingard JR, Lindemans CA, Boeckh M, Riches ML, Auletta JJ. Early cytomegalovirus reactivation remains associated with increased transplant related mortality in the current era: A CIBMTR analysis. Blood. 2016 May 19; 127(20):2427-2438. doi:10.1182/blood-2015-11-679639. Epub 2016 Feb 16. ncbi.nlm.nih.gov/pubmed/26884374







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Williams KM, Ahn KW, Chen M, Aljurf MD, Agwu AL, Chen AR, Walsh TJ, Szabolcs P, Boeckh MJ, Auletta JJ, Lindemans CA, Zanis-Neto J, Malvezzi M, Lister J, de Toledo Codina JS, Sackey K, Chakrabarty JLH, Ljungman P, Wingard JR, Seftel MD, Seo S, Hale GA, Wirk B, Smith MS, Savani BN, Lazarus HM, Marks DI, Ustun C, Abdel-Azim H, Dvorak CC, Szer J, Storek J, Yong A, Riches MR. The incidence, mortality and timing of Pneumocystis jiroveci pneumonia after hematopoietic cell transplantation: A CIBMTR analysis. Bone Marrow Transplantation. 2016 Apr 1; 51(4):573-580. doi:10.1038/bmt.2015.316. Epub 2016 Jan 4. PMC4823157. ncbi.nlm.nih.gov/pubmed/26726945

2015

Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papanicolaou GA, Wingard JR, Young J-AH, Bennett CL. Risk factors and impact of non-Aspergillus mold infections following allogeneic HCT: A CIBMTR infection and immune reconstitution analysis. Bone Marrow Transplantation. 2016 Feb 1; doi:10.1038/bmt.2015. 318. Epub 2015 Nov 2. ncbi.nlm.nih.gov/pubmed/26838373

Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papanicolaou GA, Wingard JR, Young J-AH, Bennett CL. Risk factors and impact of non-Aspergillus mold infections following allogeneic HCT: A CIBMTR infection and immune reconstitution analysis. Bone Marrow Transplantation. 2016 Feb 1; 51(2):277-282. doi:10. 1038/bmt.2015.263. Epub 2015 Nov 2. PMC4740251. ncbi.nlm.nih.gov/pubmed/26524262

10.3 Current Studies

IN16-02 Title: Determination of the burden of mucosal barrier injury-laboratory confirmed

bloodstream infections in the first 100 days after stem cell transplant PIs: Christopher Dandoy (Cincinnati Children's Hospital Medical Center) Paulina Daniels (Cincinnati Children's Hospital Medical Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) IN17-01 Title: Incidence and impact of cytomegalovirus infection in haploidentical and

matched-related donors receiving post-transplant cyclophosphamide: A CIBMTR analysis

PI: Scott Goldsmith (Washington University) Status: Analysis (as of July 1, 2019)






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IN18-01 Title: Comparison of early (By D+100) infections between posttransplantation

cyclophosphamide and other GVHD prophylaxis PIs: Genovefa Papanicolaou (Memorial Sloan Kettering Cancer Center) Celalettin Ustun (Rush University Medical Center) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) IN18-02 Title: Study the incidence and impact of C diff infection within 100 days on transplant

outcomes after allogeneic stem cell transplant PIs: Muthalagu Ramanthan (UMass Memorial Medical Center) Bipin Savani (Vanderbilt University Medical Center) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) IN19-01 Title: Immune recovery predicts post-transplant outcomes PI: Miguel-Angel Perales (Memorial Sloan Kettering Cancer Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) IN19-02 Title: Impact of antibacterial prophylaxis on outcomes after allogeneic hematopoietic

stem cell transplant PIs: C Dandoy (Cincinnati Children's Hospital Medical Center) Priscila Alonso (Cincinnati Children's Hospital Medical Center) Z El Boghdadly (The Ohio State University) Status: Draft Protocol Received (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 11.0 LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE

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11.0 LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE

11.1 Leadership

Chair: Minoo Battiwalla, MD, MS, Sarah Cannon Research Institute Email: [email protected] Chair: David Buchbinder, MD, Children's Hospital of Orange County Email: [email protected] Chair: Betty Hamilton, MD, Cleveland Clinic Taussig Cancer Institute Email: [email protected] Scientific Director: Bronwen Shaw, MBChB, MRCP, PhD, CIBMTR Milwaukee Email: [email protected] Asst Sci Director: Rachel Phelan, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Stephanie Bo-Subait, MPH, CIBMTR Minneapolis Email: [email protected]


2018

Inamoto Y, Valdés-Sanz N, Ogawa Y, Alves M, Berchicci L, Galvin J, Greinix H, Hale GA, Horn B, Kelly D, Liu H, Rowley S, Schoemans H, Shah A, Lupo Stanghellini MT, Argrawal V, Ahmed I, Ali A, Bhatt N, Byrne M, Chhabra S, DeFilipp Z, Fahnehjelm K, Farhadfar N, Horn E, Lee C, Nathan S, Penack O, Prasad P, Rotz S, Rovó A, Yared J, Pavletic S, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers MED, Shaw BE, Petriček I. Ocular graft-versus-host disease after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT. Bone Marrow Transplantation. doi:10.1038/s41409-018-0340-0. Epub 2018 Dec 7. ncbi.nlm.nih.gov/ pubmed/30531954

Inamoto Y, Petriček I, Burns L, Chhabra S, DeFilipp Z, Hematti P, Rovó A, Schears R, Shah A, Agrawal V, Al-Khinji A, Ahmed I, Ali A, Aljurf M, Alkhateeb H, Beitinjaneh A, Bhatt N, Buchbinder D, Byrne M, Callander N, Fahnehjelm K, Farhadfar N, Gale RP, Ganguly S, Hildebrandt GC, Horn E, Jakubowski A, Kamble RT, Law J, Lee C, Nathan S, Penack O, Pingali R, Prasad P, Pulanic D, Rotz S, Shreenivas A, Steinberg A, Tabbara K, Tichelli A, Wirk B, Yared J, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers MED, Shaw BE, Valdés-Sanz N. Non-GVHD ocular complications after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT. Bone Marrow Transplantation. 2019 May 1; 54(5):648-661. doi:10.1038/s41409-018-0339-6. Epub 2018 Dec 7. PMC6497536. ncbi.nlm.nih.gov/pubmed/30531955












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Inamoto Y, Petriček I, Burns L, Chhabra S, DeFilipp Z, Hematti P, Rovó A, Schears R, Shah A, Argrawal V, Ahmed A, Ahmed I, Ali A, Aljurf M, Alkhateeb H, Beitinjaneh A, Bhatt N, Buchbinder D, Byrne M, Callander N, Fahnehjelm K, Farhadfar N, Gale RP, Ganguly S, Hildebrandt GC, Horn E, Jakubowski A, Kamble RT, Law J, Lee C, Nathan S, Penack O, Pingali R, Prasad P, Pulanic D, Rotz S, Shreenivas A, Steinberg A, Tabbara K, Tichelli A, Wirk B, Yared J, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers, Shaw BE, Valdés-Sanz N. Non-GVHD ocular complications after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Transplant Complications Working Party of the EBMT. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 May 1; 25(5):e145-e154. doi:10.1016/j.bbmt.2018.11.033. Epub 2018 Dec 3. PMC6511311. ncbi.nlm.nih.gov/pubmed/30521975

Inamoto Y, Valdés-Sanz N, Ogawa Y, Alves M, Berchicci L, Galvin J, Greinix H, Hale GA, Horn B, Kelly D, Liu H, Rowley S, Schoemans H, Shah A, Lupo Stanghellini MT, Agrawal V, Ahmed I, Ali A, Bhatt N, Byrne M, Chhabra S, DeFilipp Z, Fahnehjelm K, Farhadfar N, Horn E, Lee C, Nathan S, Penack O, Prasad P, Rotz S, Rovó A, Yared J, Pavletic S, Basak GW, Battiwalla M, Duarte R, Savani BN, Flowers MED, Shaw BE, Petriček I. Ocular graft-versus-host disease after hematopoietic cell transplantation: Expert review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Transplant Complications Working Party of the European Society of Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Feb 1; 25(2):e46-e54. doi:10.1016/j.bbmt. 2018.11.021. Epub 2018 Nov 24. PMC6362842. ncbi.nlm.nih.gov/pubmed/30481594

Bhatt NS, Brazauskas R, Tecca HR, Carreras J, Burns LJ, Phelan R, Salit RB, Syrjala KL, Talano JM, Shaw BE. Post-transplantation employment status of adult survivors of childhood allogeneic hematopoietic cell transplant: A report from the Center for International Blood and Marrow Transplant Research (CIBMTR). Cancer. 2019 Jan 1; 125(1):144-152. doi:10.1002/cncr.31781. Epub 2018 Oct 12. PMC6310211. ncbi.nlm.nih.gov/pubmed/30368771

Norkin M, Shaw BE, Brazauskas R, Tecca HR, Leather HL, Gea-Banacloche J, Kamble RT, DeFilipp Z, Jacobsohn DA, Ringden O, Inamoto Y, Kasow KA, Buchbinder D, Shaw P, Hematti P, Schears R, Badawy SM, Lazarus HM, Bhatt N, Horn B, Chhabra S, Page KM, Hamilton B, Hildebrandt GC, Yared JA, Agrawal V, Beitinjaneh AM, Majhail N, Kindwall-Keller T, Olsson RF, Schoemans H, Gale RP, Ganguly S, Ahmed IA, Schouten HC, Liesveld JL, Khera N, Steinberg A, Shah AJ, Solh M, Marks DI, Rybka W, Aljurf M, Dietz AC, Gergis U, George B, Seo S, Flowers MED, Battiwalla M, Savani BN, Riches ML, Wingard JR. Characteristics of late fatal infections after allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Feb 1; 25(2):362-368. doi:10.1016/j.bbmt. 2018.09.031. Epub 2018 Oct 2. PMC6339825. ncbi.nlm.nih.gov/pubmed/30287390






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Radivoyevitch T, Dean RM, Shaw BE, Brazauskas R, Tecca HR, Molenaar RJ, Battiwalla M, Savani BN, Flowers MED, Cooke KR, Hamilton BK, Kalaycio M, Maciejewski JP, Ahmed I, Akpek G, Bajel A, Buchbinder D, Cahn J-Y, D'Souza A, Daly A, DeFilipp Z, Ganguly S, Hamadani M, Hayashi RJ, Hematti P, Inamoto Y, Khera N, Kindwall-Keller T, Landau H, Lazarus H, Majhail NS, Marks DI, Olsson RF, Seo S, Steinberg A, William BM, Wirk B, Yared JA, Aljurf M, Abidi MH, Allewelt H, Beitinjaneh A, Cook R, Cornell RF, Fay JW, Hale G, Chakrabarty JH, Jodele S, Kasow KA, Mahindra A, Malone AK, Popat U, Rizzo JD, Schouten HC, Warwick AB, Wood WA, Sekeres MA, Litzow MR, Gale RP, Hashmi SK. Risk of acute myeloid leukemia and myelodysplastic syndrome after autotransplants for lymphomas and plasma cell myeloma. Leukemia Research. 2018 Nov 1; 74:130-136. doi:10.1016/j.leukres.2018.07.016. Epub 2018 Jul 19. PMC6219911. ncbi.nlm.nih.gov/pubmed/30055822

Bhatt NS, Brazauskas R, Tecca HR, Vogel J, Mattila D, Lee SJ, Horowitz MM, Rizzo JD, Shaw BE. Female sex is associated with poor health-related quality of life in children at 12 months post-hematopoietic cell transplantation. Journal of Pediatric Hematology/Oncology. doi:10.1097/MPH.0000000000001239. Epub 2018 Jun 19. ncbi.nlm.nih.gov/pubmed/29923857

Duncan CN, Brazauskas R, Huang J, Shaw BE, Majhail NS, Savani BN, Flowers MED, Battiwalla M, Beebe K, Dietz AC, Dvorak CC, Giller R, Jacobsohn DA, Kletzel M, Martin PL, Nemecek ER, Nuechterlein B, Talano J-A, Pulsipher MA, Baker KS. Late cardiovascular morbidity and mortality following pediatric allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. doi:10.1038/s41409-018-0155-z. Epub 2018 Mar 26. ncbi.nlm.nih.gov/ pubmed/29581480

D'Souza A, Millard H, Knight J, Brazauskas R, Lee SJ, Flynn KE, Rizzo JD, Shaw BE. Prevalence of self-reported sleep dysfunction before allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. doi:10.1038/s41409-018-0150-4. Epub 2018 Mar 7. ncbi.nlm.nih.gov/ pubmed/29515251

Buchbinder D, Kelly DL, Duarte RF, Auletta JJ, Bhatt N, Byrne M, DeFilipp Z, Gabriel M, Mahindra A, Norkin M, Schoemans H, Shah AJ, Ahmed I, Atsuta Y, Basak GW, Beattie S, Bhella S, Bredeson C, Bunin N, Dalal J, Daly A, Gajewski J, Gale RP, Galvin J, Hamadani M, Hayashi RJ, Adekola K, Law J, Lee CJ, Liesveld J, Malone AK, Nagler A, Naik S, Nishihori T, Parsons SK, Scherwath A, Schofield HL, Soiffer R, Szer J, Twist I, Warwick AB, Wirk BM, Yi J, Battiwalla M, Flowers MDE, Savani B, Shaw BE. Neurocognitive dysfunction in hematopoietic cell transplant recipients: Expert review from the Late Effects and Quality of Life Working Committee of the CIBMTR and Complications and Quality of Life Working Party of the EBMT. Bone Marrow Transplantation. 2018 May 1; 53(5):535-555. doi:10.1038/s41409-017-0055-7. Epub 2018 Jan 17. PMC5985976. ncbi.nlm.nih.gov/pubmed/29343837









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2017

Myers RM, Hill BT, Shaw BE, Kim S, Millard HR, Battiwalla M, Majhail NS, Buchbinder D, Lazarus HM, Savani BN, Flowers MED, D'Souza A, Ehrhardt MJ, Langston A, Yared JA, Hayashi RJ, Daly A, Olsson RF, Inamoto Y, Malone AK, DeFilipp Z, Margossian SP, Warwick AB, Jaglowski S, Beitinjaneh A, Fung H, Kasow KA, Marks DI, Reynolds J, Stockerl-Goldstein K, Wirk B, Wood WA, Hamadani M, Satwani P. Long-term outcomes among 2-year survivors of autologous hematopoietic cell transplantation for Hodgkin and diffuse large B-cell lymphoma. Cancer. 2018 Feb 15; 124(4):816-825. doi:10.1002/cncr.31114. Epub 2017 Nov 10. PMCID5871233. ncbi.nlm.nih.gov/pubmed/29125192

Shaw BE, Syrjala KL, Onstad LE, Chow EJ, Flowers ME, Jim H, Baker KS, Buckley S, Fairclough DL, Horowitz MM, Lee SJ. PROMIS measures can be used to assess symptoms and function in long-term hematopoietic cell transplantation survivors. Cancer. 2018 Feb 15; 124(4):841-849. doi:10.1002/cncr.31089. Epub 2017 Oct 26. PMC5800994. ncbi.nlm.nih.gov/pubmed/ 29072787

Kelly DL, Buchbinder D, Duarte RF, Auletta JJ, Bhatt N, Byrne M, DeFilipp Z, Gabriel M, Mahindra A, Norkin M, Schoemans H, Shah AJ, Ahmed I, Atsuta Y, Basak GW, Beattie S, Bhella S, Bredeson C, Bunin N, Dalal J, Daly A, Gajewski J, Gale RP, Galvin J, Hamadani M, Hayashi RJ, Adekola K, Law J, Lee CJ, Liesveld J, Malone AK, Nagler A, Naik S, Nishihori T, Parsons SK, Scherwath A, Schofield H-L, Soiffer R, Szer J, Twist I, Warwick A, Wirk BM, Yi J, Battiwalla M, Flowers ME, Savani B, Shaw BE. Neurocognitive dysfunction in hematopoietic cell transplant recipients: Expert review from the Late Effects and Quality of Life Working Committee of the Center for International Blood and Marrow Transplant Research and Complications and Quality of Life Working Party of the European Society for Blood and Marrow Transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Feb 1; 24(2):228-241. doi:10.1016/j.bbmt.2017.09.004. Epub 2017 Sep 20. PMC5768142. ncbi.nlm.nih.gov/pubmed/28939455

Shaw BE, Brazauskas R, Millard HR, Fonstad R, Flynn KE, Abernethy A, Vogel J, Petroske C, Mattila D, Drexler R, Lee SJ, Horowitz MM, Rizzo JD. Centralized patient-reported outcome data collection in transplantation is feasible and clinically meaningful. Cancer. 2017 Dec 1; 123(23):4687-4700. doi:10.1002/cncr.30936. Epub 2017 Aug 17. PMC5693638. ncbi.nlm.nih.gov/pubmed/28817182

Vrooman LM, Millard HR, Brazauskas R, Majhail NS, Battiwalla M, Flowers ME, Savani BN, Akpek G, Aljurf M, Bajwa R, Baker KS, Beitinjaneh A, Bitan M, Buchbinder D, Chow E, Dietz AC, Diller L, Gale RP, Hashmi SK, Hayashi RJ, Hematti P, Kamble RT, Kasow KA, Kletzel M, Lazarus HM, Malone AK, Marks DI, O'Brien TA, Olsson RF, Ringden O, Seo S, Steinberg A, Yu LC, Warwick A, Shaw B, Duncan C. Survival and late effects after allogeneic hematopoietic cell transplantation for hematologic malignancy at less than three years of age. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Aug 1; 23(8):1327-1334. doi:10.1016/j.bbmt.2017.04.017. Epub 2017 Apr 28. PMC5666571. ncbi.nlm.nih.gov/pubmed/28461213








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Gabriel M, Shaw B, Brazauskas R, Chen M, Margolis DA, Sengelov H, Dahlberg A, Ahmed IA, Delgado D, Lazarus HM, Gibson B, Myers KC, Kamble RT, Abdel-Mageed A, Li C-K, Flowers MED, Battiwalla M, Savani BN, Majhail N, Shaw P. Risk factors for subsequent central nervous system tumors in pediatric allogeneic hematopoietic cell transplant: A study from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Aug 1; 23(8):1320-1326. doi:10.1016/j.bbmt.2017.04.004. Epub 2017 Apr 12. PMC5518678. ncbi.nlm.nih.gov/pubmed/28411175

2016

Hashmi SK, Bredeson C, Duarte RF, Farnia S, Ferrey S, Fitzhugh C, Flowers MED, Gajewski J, Gastineau D, Greenwald M, Jagasia M, Martin P, Rizzo JD, Schmit-Pokorny K, Majhail NS. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 May 1; 23(5):717-725. doi:10.1016/j.bbmt.2016.09.025. Epub 2016 Oct 3. PMC5378687. ncbi.nlm.nih.gov/ pubmed/27713091

Morton LM, Saber W, Baker KS, Barrett AJ, Bhatia S, Engels EA, Gadalla SM, Kleiner DE, Pavletic S, Burns LJ. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Subsequent Neoplasms Working Group Report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Mar 1; 23(3):367-378. doi:10.1016/j.bbmt.2016.09.005. Epub 2016 Sep 12. PMC5285307. ncbi.nlm.nih.gov/pubmed/27634019

Shaw BE, Hahn T, Martin PJ, Mitchell SA, Petersdorf EW, Armstrong GT, Shelburne N, Storer BE, Bhatia S. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Research Methodology and Study Design Working Group report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):10-23. doi:10.1016/j.bbmt.2016.08.018. Epub 2016 Aug 30. PMC5182148. ncbi.nlm.nih.gov/pubmed/27590102

DeFilipp Z, Duarte RF, Snowden JA, Majhail NS, Greenfield DM, Miranda JL, Arat M, Baker KS, Burns LJ, Duncan CN, Gilleece M, Hale GA, Hamadani M, Hamilton BK, Hogan WJ, Hsu JW, Inamoto Y, Kamble RT, Lupo-Stanghellini MT, Malone AK, McCarthy P, Mohty M, Norkin M, Paplham P, Ramanathan M, Richart JM, Salooja N, Schouten HC, Schoemans H, Seber A, Steinberg A, Wirk BM, Wood WA, Battiwalla M, Flowers MED, Savani BN, Shaw BE. Metabolic syndrome and cardiovascular disease following hematopoietic cell transplantation: Screening and preventive practice recommendations from CIBMTR and EBMT. Bone Marrow Transplantation. 2017 Feb 1; 52(2):173-182. doi:10.1038/bmt.2016.203. Epub 2016 Aug 22. PMC5288134. ncbi.nlm.nih.gov/pubmed/27548466








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DeFilipp Z, Duarte RF, Snowden JA, Majhail NS, Greenfield DM, Miranda JL, Arat M, Baker KS, Burns LJ, Duncan CN, Gilleece M, Hale GA, Hamadani M, Hamilton BK, Hogan WJ, Hsu JW, Inamoto Y, Kamble RT, Lupo-Stanghellini MT, Malone AK, McCarthy P, Mohty M, Norkin M, Paplham P, Ramanathan M, Richart JM, Salooja N, Schouten HC, Schoemans H, Seber A, Steinberg A, Wirk BM, Wood WA, Battiwalla M, Flowers MED, Savani BN, Shaw BE. Metabolic syndrome and cardiovascular disease following hematopoietic cell transplantation: Screening and preventive practice recommendations from CIBMTR and EBMT. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.05.007. Epub 2016 May 14. ncbi.nlm.nih.gov/ pubmed/27184625

Shaw BE, Lee SJ, Horowitz MM, Wood WA, Rizzo JD, Flynn KE. Can we agree on patient-reported outcome measures for assessing hematopoietic cell transplantation patients? A study from the CIBMTR and BMT CTN. Bone Marrow Transplantation. 2016 Sep 1; 51(9):1173-1179. doi:10.1038/bmt.2016.113. Epub 2016 May 9. ncbi.nlm.nih.gov/pubmed/27159181

Saber W, Zhang M-J, Steinert P, Chen M, Horowitz MM. The impact of palifermin use on hematopoietic cell transplant outcomes in children. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1460-1466. doi:10.1016/j.bbmt.2016.04.008. Epub 2016 May 7. PMC4949099. ncbi.nlm.nih.gov/pubmed/27090960

Li Z, Rubinstein SM, Thota R, Savani M, Brissot E, Shaw BE, Majhail NS, Mohty M, Savani BN. Immune-mediated complications after hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1368-1375. doi:10.1016/j.bbmt.2016.04.005. Epub 2016 Apr 14. ncbi.nlm.nih.gov/pubmed/27095688

2015

Ehrhardt MJ, Brazauskas R, He W, Rizzo JD, Shaw BE. Survival of patients who develop solid tumors following hematopoietic stem cell transplantation. Bone Marrow Transplantation. 2016 Jan 1; 51(1):83-88. doi:10.1038/bmt.2015.203. Epub 2015 Sep 14. PMC4570237. ncbi.nlm.nih.gov/pubmed/26367222

Inamoto Y, Shah NN, Savani BN, Shaw BE, Abraham AA, Ahmed IA, Akpek G, Atsuta Y, Baker KS, Basak GW, Bitan M, DeFilipp Z, Gregory TK, Greinix HT, Hamadani M, Hamilton BK, Hayashi RJ, Jacobsohn DA, Kamble RT, Kasow KA, Khera N, Lazarus HM, Malone AK, Lupo-Stanghellini MT, Margossian SP, Muffly LS, Norkin M, Ramanathan M, Salooja N, Schoemans H, Wingard JR, Wirk B, Wood WA, Yong A, Duncan CN, Flowers MED, Majhail NS. Secondary solid cancer screening following hematopoietic cell transplantation. Bone Marrow Transplantation. 2015 Aug 1; 50(8):1013-1023. doi:10.1038/bmt.2015.63. Epub 2015 Mar 30. ncbi.nlm.nih.gov/pubmed/ 25822223










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2014

Duncan CN, Majhail NS, Brazauskas R, Wang Z, Cahn J-Y, Frangoul HA, Hayashi RJ, Hsu JW, Kamble RT, Kasow KA, Khera N, Lazarus HM, Loren AW, Marks DI, Maziarz RT, Mehta P, Myers KC, Norkin M, Pidala JA, Porter DL, Reddy V, Saber W, Savani BN, Schouten HC, Steinberg A, Wall DA, Warwick AB, Wood WA, Yu LC, Jacobsohn DA, Sorror ML. Long-term survival and late effects among one-year survivors of second allogeneic hematopoietic cell transplantation for relapsed acute leukemia and myelodysplastic syndromes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jan 1; 21(1):151-158. doi:10.1016/j.bbmt.2014.10.006. Epub 2014 Oct 12. PMC4272862. ncbi.nlm.nih.gov/pubmed/25316109

Ringdén O, Brazauskas R, Wang Z, Ahmed I, Atsuta Y, Buchbinder D, Burns LJ, Cahn J-Y, Duncan C, Hale GA, Halter J, Hayashi RJ, Hsu JW, Jacobsohn DA, Kamble RT, Kamani NR, Kasow KA, Khera N, Lazarus HM, Loren AW, Marks DI, Myers KC, Ramanathan M, Saber W, Savani BN, Schouten HC, Socie G, Sorror ML, Steinberg A, Popat U, Wingard JR, Mattsson J, Majhail NS. Second solid cancers after allogeneic hematopoietic cell transplantation using reduced-intensity conditioning. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1777-1784. doi:10.1016/j.bbmt.2014. 07.009. Epub 2014 Jul 17. PMC4194257. ncbi.nlm.nih.gov/pubmed/25042734


LE12-03 Title: Solid organ transplantation and hematopoietic cell transplantation PIs: Meera Gupta (Abramson Cancer Center University of Pennsylvania Medical Center) Peter L Abt (Abramson Cancer Center University of Pennsylvania Medical Center) Matthew Levine (Abramson Cancer Center University of Pennsylvania Medical Center) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with United Network for Organ Sharing LE13-02 Title: Risk factors for melanoma following allogeneic hematopoietic stem cell

transplantation PIs: Megan Herr (Roswell Park Cancer Institute) Lindsay Morton (National Cancer Institute) Eric Engels (National Cancer Institute) Margaret Tucker (National Cancer Institute) Rochelle Curtis (NIH-Experimental Transplantation and Immunology Branch) Ruth Pfeiffer (National Cancer Institute) David A Jacobson (Vanderbilt Univeristy Medical Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with National Institutes of Health




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LE16-02 Title: An investigation of new malignant neoplasms in pediatric patients undergoing

allogeneic hematopoietic stem cell transplantation for non-malignant diseases PIs: Justine Kahn (Morgan Stanley Children’s Hospital of New York Presbyterian – Columbia

University Medical Center) Prakash Satwani (New York Presbyterian – Columbia University Medical Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) LE17-01 Title: Long-term follow up after hematopoietic stem cell transplantation for sickle cell

disease PIs: Elizabeth Stenger (Children's Healthcare of Atlanta at Egleston) Rachel Phelan (Medical College of Wisconsin) Shalini Shenoy (Washington University / St. Louis Children's Hospital) Lakshmanan Krishnamurti (Children's Healthcare of Atlanta at Egleston) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Emory University LE17-02 Title: Comparison of late effects among allogeneic hematopoietic cell transplantation

survivors conditioned with high dose total body irradiation (TBI) versus non-TBI based ablative regimens in adolescents and young adults (15-39yo) with acute leukemia

PIs: Catherine Lee (Utah Blood and Marrow Transplant Program) Lori Muffly (Stanford Health Care) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LE18-01 Title: Survival trends amongst two-year survivors of alloHCT PIs: Prakash Satwani (New York Presbyterian Hospital/Columbia University Medical

Center) Larisa Brogile (Columbia University) Status: Protocol Development (as of July 1, 2019)

Submitted (expected by June 30, 2020) LE18-02 Title: Return to work or school status in survivors of adolescent and young adult

alloHCT PIs: Neel Bhatt (Fred Hutchinson Cancer Research Center) Rachel Salit (Fred Hutchinson Cancer Research Center) Status: Manuscript Preparation (as of July 1, 2019)



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LE18-03 Title: Incorporating patient reported outcomes into individualized prognostication

tools for survival and quality of life in transplant patients PI: Bronwen Shaw (Medical College of Wisconsin) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with The Emmes Company LE19-01 Title: Long-term survival and late effects in critically ill pediatric hematopoietic cell

transplant patients PIs: Matt Zinter (University of California San Francisco Medical Center) Chris Dvorak (University of California San Francisco Medical Center) Christy Duncan (Dana Farber Cancer Institute & Boston Children's Hospital) Status: Draft Protocol Received (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with Virtual Pediatric Systems LE19-02 Title: Incidence and predictors of Long term toxicities and late side effects in elderly

patients (>=60 years) receiving allogeneic hematopoietic cell transplantation for hematological malignancies.

PIs: Muthu Veeraputhiran (University of Arkansas for Medical Sciences) Sai Ravi Pingali (M.D. Anderson Cancer Center) Akash Mukherjee (University of Arkansas for Medical Sciences) Lori Muffly (Stanford Health Care) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) LE99-01 Title: Quality of life in late HCT survivors PI: John Wingard (Shands HealthCare & University of Florida) Status: Ongoing (as of July 1, 2019)

Ongoing (expected by June 30, 2020) * Collaborative study with Univeristy of Florida SC09-05d Title: Regret having bone marrow transplant PI: Rachel Cusatis (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with University of Minnesota

2019 CIBMTR Scientific Working Committee Research Portfolio 12.0 LYMPHOMA WORKING COMMITTEE

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12.0 LYMPHOMA WORKING COMMITTEE

12.1 Leadership

Chair: Timothy Fenske, MD, MS, Froedtert Hospital Email: [email protected] Chair: Mohamed Kharfan-Dabaja, MD, MBA, Mayo Clinic Florida Email: [email protected] Chair: Craig Sauter, MD, Memorial Sloan Kettering Cancer Center - Adults Email: [email protected] Scientific Director: Mehdi Hamadani, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Kwang Woo Ahn, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Andrew St. Martin, MS, CIBMTR Milwaukee Email: [email protected]


2019

Ahmed S, Kanakry JA, Ahn KW, Litovich C, Abdel-Azim H, Aljurf M, Bacher VU, Bejanyan N, Cohen JB, Farooq U, Fuchs EJ, Bolaños-Meade J, Ghosh N, Herrera AF, Hossain NM, Inwards D, Kanate AS, Martino R, Munshi PN, Murthy H, Mussetti A, Nieto Y, Perales M-A, Romee R, Savani BN, Seo S, Wirk B, Yared JA, Sureda A, Fenske TS, Hamadani M. Lower GVHD and relapse risk in PTCy-based haploidentical vs matched sibling donor RIC transplant for Hodgkin lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.05.025. Epub 2019 May 24. PMC6553842. ncbi.nlm.nih.gov/pubmed/31132455

Kanate AS, Kumar A, Dreger P, Dreyling M, Le Gouill S, Corradini, Bredeson C, Fenske TS, Smith SM, Sureda A, Moskowitz A, Friedberg JW, Inwards DJ, Herrera AF, Kharfan-Dabaja MA, Reddy N, Montoto S, Robinson S, Abutalib SA, Gisselbre C, Vose J, Gopal A, Shadman M, Perales M-A, Carpenter P, Savani BN, Hamadani M. Maintenance therapies for Hodgkin and non-Hodgkin lymphomas after autologous transplantation: A consensus project of ASBMT, CIBMTR, and the Lymphoma Working Party of EBMT. JAMA Oncology. doi:10.1001/jamaoncol.2018.6278. Epub 2019 Feb 28. ncbi.nlm.nih.gov/pubmed/30816957

Dreger P, Sureda A, Ahn KW, Eapen M, Litovich C, Fine H, Boumendil A, Gopal A, Herrera AF, Schmid C, Diez-Martin JL, Fuchs E, Bolaños -Meade J, Gooptu M, Al Malki MM, Castagna L, Ciurea SO, Dominietto A, Blaise D, Ciceri F, Tischer J, Corradini P, Montoto S, Robinson S, GÃ¼lbas Z, Hamadani M. PTCy-based haploidentical vs matched related or unrelated donor reduced-intensity conditioning transplant for DLBCL. Blood Advances. 2019 Feb 12; 3(3):360-369. doi:10.1182/bloodadvances.2018027748. Epub 2019 Feb 5. PMC6373757. ncbi.nlm.nih.gov/ pubmed/30723110












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Epperla N, Ahn KW, Litovich C, Ahmed S, Battiwalla M, Cohen JB, Dahi P, Farhadfar N, Farooq U, Freytes CO, Ghosh N, Haverkos B, Herrera A, Hertzberg M, Hildebrandt G, Inwards D, Kharfan-Dabaja MA, Khimani F, Lazarus H, Lazaryan A, Lekakis L, Murthy H, Nathan S, Nishihori T, Pawarode A, Prestidge T, Ramakrishnan P, Rezvani AR, Romee R, Shah NN, Sureda A, Fenske TS, Hamadani M. Allogeneic hematopoietic cell transplantation provides effective salvage despite refractory disease or failed prior autologous transplant in angioimmunoblastic T-cell lymphoma: A CIBMTR analysis. Journal of Hematology & Oncology. 12(1):6. doi:10.1186/ s13045-018-0696-z. Epub 2019 Jan 10. PMC6329157. ncbi.nlm.nih.gov/pubmed/30630534

2018

Sureda A, Montoto S, Dreger P, Hamadani M, Pasquini MC. Reply to The persistent uncertainty of when to recommend allogeneic stem cell transplantation in follicular lymphoma. Cancer. doi:10.1002/cncr.31564. Epub 2018 Jun 7. ncbi.nlm.nih.gov/pubmed/29878309

Shah NN, Ahn KW, Litovich C, Fenske TS, Ahmed S, Battiwalla M, Bejanyan N, Dahi PB, Bolaños-Meade J, Chen AI, Ciurea SO, Bachanova V, DeFilipp Z, Epperla N, Farhadfar N, Herrera AF, Haverkos BM, Holmberg L, Hossain NM, Kharfan-Dabaja MA, Kenkre VP, Lazarus HM, Murthy HS, Nishihori T, Rezvani AR, D'Souza A, Savani BN, Ulrickson ML, Waller EK, Sureda A, Smith SM, Hamadani M. Outcomes of Medicare-age eligible NHL patients receiving RIC allogeneic transplantation: A CIBMTR analysis. Blood Advances. 2018 Apr 24; 2(8):933-940. doi:10.1182/bloodadvances.2018018531. Epub 2018 Apr 23. PMC5916010. ncbi.nlm.nih.gov/ pubmed/29685953

Smith SM, Godfrey J, Ahn KW, DiGilio A, Ahmed S, Agrawal V, Bachanova V, Bacher U, Bashey A, Bolaños-Meade J, Cairo M, Chen A, Chhabra S, Copelan E, Dahi PB, Aljurf M, Farooq U, Ganguly S, Hertzberg M, Holmberg L, Inwards D, Kanate AS, Karmali R, Kenkre VP, Kharfan-Dabaja MA, Klein A, Lazarus HM, Mei M, Mussetti A, Nishihori T, Ramakrishnan Geethakumari P, Saad A, Savani BN, Schouten HC, Shah N, Urbano-Ispizua A, Vij R, Vose J, Sureda A, Hamadani M. Autologous transplantation versus allogeneic transplantation in patients with follicular lymphoma experiencing early treatment failure. Cancer. 2018 Jun 15; 124(12):2541-2551. doi:10.1002/cncr.31374. Epub 2018 Apr 12. PMC5990449. ncbi.nlm.nih.gov/pubmed/29645093

Sureda A, Zhang M-J, Dreger P, Carreras J, Fenske T, Finel H, Schouten H, Montoto S, Robinson S, Smith SM, Boumedil A, Hamadani M, Pasquini MC. Allogeneic hematopoietic stem cell transplantation for relapsed follicular lymphoma: A combined analysis on behalf of the Lymphoma Working Party of the EBMT and the Lymphoma Committee of the CIBMTR. Cancer. 2018 Apr 15; 124(8):1733-1742. doi:10.1002/cncr.31264. Epub 2018 Feb 9. PMC5946312. ncbi.nlm.nih.gov/pubmed/29424927








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2017

Casulo C, Friedberg JW, Ahn KW, Flowers C, DiGilio A, Smith SM, Ahmed S, Inwards D, Aljurf M, Chen AI, Choe H, Cohen J, Copelan E, Farooq U, Fenske TS, Freytes C, Gaballa S, Ganguly S, Jethava Y, Kamble RT, Kenkre VP, Lazarus H, Lazaryan A, Olsson RF, Rezvani AR, Rizzieri D, Seo S, Shah GL, Shah N, Solh M, Sureda A, William B, Cumpston A, Zelenetz AD, Link BK, Hamadani M. Autologous transplantation in follicular lymphoma with early therapy failure: A National LymphoCare Study and Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jun 1; 24(6):1163-1171. doi:10.1016/j.bbmt.2017.12. 771. Epub 2017 Dec 11. PMC5993598. ncbi.nlm.nih.gov/pubmed/29242111

Hamadani M, Horowitz MM. Allogeneic transplantation for follicular lymphoma: Does one size fit all? Journal of Oncology Practice. 2017 Dec 1; 13(12):798-806. doi:10.1200/JOP.2017. 026336. Epub 2017 Dec 1. PMC5728364. ncbi.nlm.nih.gov/pubmed/29232542

Epperla N, Ahn KW, Armand P, Jaglowski S, Ahmed S, Kenkre VP, Savani B, Jagasia M, Shah NN, Fenske TS, Sureda A, Smith SM, Hamadani M. Fludarabine and busulfan versus fludarabine, cyclophosphamide, and rituximab as reduced-intensity conditioning for allogeneic transplantation in follicular lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Jan 1; 24(1):78-85. doi:10.1016/j.bbmt.2017.10.011. Epub 2017 Oct 13. PMC5743624. ncbi.nlm.nih.gov/pubmed/ 29032272

Kanate AS, DiGilio A, Ahn KW, Al Malki M, Jacobsen E, Steinberg A, Hammerschlak N, Kharfan-Dabaja M, Salit R, Ball E, Bashir Q, Cashen A, Couriel D, Diez-Martin J, Katsanis E, Linhares Y, Mori S, Nash R, Pawarode A, Perales M-A, Phipps CD, Richman C, Savani BN, Shapira MY, Stiff P, Strair R, Fenske TS, Smith SM, Sureda A, Olteanu H, Hamadani M. Allogeneic haematopoietic cell transplantation for extranodal natural killer / T-cell lymphoma, nasal type: A CIBMTR analysis. British Journal of Haematology. doi:10.1111/bjh.14879. Epub 2017 Aug 2. PMC5796874. ncbi.nlm.nih.gov/pubmed/28771676

Epperla N, Ahn KW, Ahmed S, Jagasia M, DiGilio A, Devine SM, Jaglowski S, Kennedy V, Rezvani AR, Smith SM, Sureda A, Fenske TS, Kharfan-Dabaja MA, Armand P, Hamadani M. Rituximab-containing reduced-intensity conditioning improves progression-free survival following allogeneic transplantation in B cell non-Hodgkin lymphoma. Journal of Hematology & Oncology. 2017 Jun 12; 10(1):117. doi:10.1186/s13045-017-0487-y. Epub 2017 Jun 12. PMC5469142. ncbi.nlm.nih.gov/pubmed/28606176

Hamadani M, Kanate AS, DiGilio A, Ahn KW, Smith SM, Lee JW, Ayala E, Chao N, Hari P, Bolaños-Meade J, Gress R, Smedegaard Anderson N, Chen Y-B, Farooq U, Schiller G, Yared J, Sureda A, Fenske TS, Olteanu H. Allogeneic hematopoietic cell transplantation for aggressive NK cell leukemia. A Center for International Blood and Marrow Transplant Research analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 May 1; 23(5):853-856. doi:10.1016/j.bbmt.2017.01.082. Epub 2017 Feb 1. PMC5410937. ncbi.nlm.nih.gov/pubmed/28161608









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2016

Ghosh N, Karmali R, Rocha V, Ahn KW, DiGilio A, Hari PN, Bachanova V, Bacher U, Dahi P, de Lima M, D'Souza A, Fenske TS, Ganguly S, Kharfan-Dabaja MA, Prestidge TD, Savani BN, Smith SM, Sureda A, Waller EK, Jaglowski S, Herrera AF, Armand P, Salit RB, Wagner-Johnston ND, Fuchs E, Bolaños-Meade J, Hamadani M. Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLA-matched sibling donors. Journal of Clinical Oncology. doi:10.1200/JCO.2015.66.3476. Epub 2016 Jun 6. PMC5012706. ncbi.nlm.nih.gov/ pubmed/27269951

Fenske TS, Hamadani M, Cohen JB, Costa LJ, Kahl BS, Evens AM, Hamlin PA, Lazarus HM, Petersdorf E, Bredeson C. Allogeneic hematopoietic cell transplantation as curative therapy for patients with non-Hodgkin lymphoma: Increasingly successful application to older patients. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Sep 1; 22(9):1543-1551. doi:10.1016/ j.bbmt.2016.04.019. Epub 2016 Apr 27. PMC4981559. ncbi.nlm.nih.gov/pubmed/27131863

Flowers CR, Costa LJ, Pasquini MC, Le-Rademacher J, Lill M, Shore TB, Vaughan W, Craig M, Freytes CO, Shea TC, Horwitz ME, Fay JW, Mineishi S, Rondelli D, Mason J, Braunschweig I, Ai W, Yeh RF, Rodriguez TE, Flinn I, Comeau T, Yeager AM, Pulsipher MA, Bence-Bruckler I, Laneuville P, Bierman P, Chen AI, Kato K, Wang Y, Xu C, Smith AJ, Waller EK. Efficacy of pharmacokinetics-directed busulfan, cyclophosphamide and etoposide conditioning and autologous stem cell transplantation for lymphoma: Comparison of a multicenter Phase 2 study and CIBMTR outcomes. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2016.03.018. Epub 2016 Mar 31. PMC4914052. ncbi.nlm.nih.gov/pubmed/27040394

Fenske TS, Ahn KW, Graff TM, DiGilio A, Bashir Q, Kamble RT, Ayala E, Bacher U, Brammer JE, Cairo M, Chen A, Chen Y-B, Chhabra S, D'Souza A, Farooq U, Freytes C, Ganguly S, Hertzberg M, Inwards D, Jaglowski S, Kharfan-Dabaja MA, Lazarus HM, Nathan S, Pawarode A, Perales M-A, Reddy N, Seo S, Sureda A, Smith SM, Hamadani M. Allogeneic transplantation provides durable remission in a subset of DLBCL patients relapsing after autologous transplantation. British Journal of Haematology. doi:10.1111/bjh.14046. Epub 2016 Mar 15. PMC4940282. ncbi.nlm.nih.gov/pubmed/26989808

2015

Kanate AS, Mussetti A, Kharfan-Dabaja MA, Ahn KW, DiGilio A, Beitinjaneh A, Chhabra S, Fenske TS, Freytes C, Gale RP, Ganguly S, Hertzberg M, Klyuchnikov E, Lazarus HM, Olsson R, Perales M-A, Rezvani A, Riches M, Saad A, Slavin S, Smith SM, Sureda A, Yared J, Ciurea S, Armand P, Salit R, Bolaños-Meade J, Hamadani M. Reduced-intensity transplantation for lymphomas using haploidentical related donor’s vs HLA-matched unrelated donors. Blood. 2016 Feb 18; 127(7):938-947. doi:10.1182/blood-2015-09-671834. Epub 2015 Dec 15. PMC4760094. ncbi.nlm.nih.gov/pubmed/26670632








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Klyuchnikov E, Bacher U, Woo Ahn K, Carreras J, Kröger NM, Hari PN, Ku GH, Ayala E, Chen AI, Chen Y-B, Cohen JB, Freytes CO, Gale RP, Kamble RT, Kharfan-Dabaja MA, Lazarus HM, Martino R, Mussetti A, Savani BN, Schouten HC, Usmani SZ, Wiernik PH, Wirk B, Smith SM, Sureda A, Hamadani M. Long-term survival outcomes of reduced-intensity allogeneic or autologous transplantation in relapsed grade 3 follicular lymphoma. Bone Marrow Transplantation. 2016 Jan 1; 51(1):58-66. doi:10.1038/bmt.2015.223. Epub 2015 Oct 5. PMC4703480. ncbi.nlm.nih.gov/pubmed/26437062

Klyuchnikov E, Bacher U, Kröger NM, Hari PN, Ahn KW, Carreras J, Bachanova V, Bashey A, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Hertzberg MS, Holmberg LA, Kharfan-Dabaja MA, Klein A, Ku GH, Laport GG, Lazarus HM, Miller AM, Mussetti A, Olsson RF, Slavin S, Usmani SZ, Vij R, Wood WA, Maloney DG, Sureda AM, Smith SM, Hamadani M. Reduced-intensity allografting as first transplantation approach in relapsed/refractory grades one and two follicular lymphoma provides improved outcomes in long-term survivors. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2091-2099. doi:10.1016/j.bbmt.2015.07.028. Epub 2015 Aug 4. PMC4639453. ncbi.nlm.nih.gov/pubmed/26253007

Satwani P, Ahn KW, Carreras J, Abdel-Azim H, Cairo MS, Cashen A, Chen AI, Cohen JB, Costa LJ, Dandoy C, Fenske TS, Freytes CO, Ganguly S, Gale RP, Ghosh N, Hertzberg MS, Hayashi RJ, Kamble RT, Kanate AS, Keating A, Kharfan-Dabaja MA, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Prestidge TD, Rolon JM, Savani BN, Vose JM, Wood WA, Inwards DJ, Bachanova V, Smith SM, Maloney DG, Sureda A, Hamadani M. A prognostic model predicting autologous transplantation outcomes in children, adolescents and young adults with Hodgkin lymphoma. Bone Marrow Transplantation. 2015 Nov 1; 50(11):1416-1423. doi:10.1038/ bmt.2015.177. Epub 2015 Aug 3. PMC4633349. ncbi.nlm.nih.gov/pubmed/26237164

Urbano-Ispizua A, Pavletic SZ, Flowers ME, Klein JP, Zhang M-J, Carreras J, Montoto S, Perales M-A, Aljurf MD, Akpek G, Bredeson CN, Costa LJ, Dandoy C, Freytes CO, Fung HC, Gale RP, Gibson J, Hamadani M, Hayashi RJ, Inamoto Y, Inwards DJ, Lazarus HM, Maloney DG, Martino R, Munker R, Nishihori T, Olsson RF, Rizzieri DA, Reshef R, Saad A, Savani BN, Schouten HC, Smith SM, Socié G, Wirk B, Yu LC, Saber W. The impact of graft-versus-host disease on the relapse rate in patients with lymphoma depends on the histological subtype and the intensity of the conditioning regimen. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1746-1753. doi:10.1016/ j.bbmt.2015.05.010. Epub 2015 May 15. PMC4568162. ncbi.nlm.nih.gov/pubmed/25981509

Bachanova V, Burns LJ, Ahn KW, Laport GG, Akpek G, Kharfan-Dabaja MA, Nishihori T, Agura E, Armand P, Jaglowski SM, Cairo MS, Cashen AF, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Ghosh N, Holmberg LA, Inwards DJ, Kanate AS, Lazarus HM, Malone AK, Munker R, Mussetti A, Norkin M, Prestidge TD, Rowe JM, Satwani P, Siddiqi T, Stiff PJ, William BM, Wirk B, Maloney DG, Smith SM, Sureda AM, Carreras J, Hamadani M. Impact of pretransplantation 18F-fluorodeoxy glucose-positron emission tomography status on outcomes after allogeneic hematopoietic cell transplantation for non-Hodgkin lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1605-1611. doi:10.1016/j.bbmt.2015.05.007. Epub 2015 May 14. PMC4558181. ncbi.nlm.nih.gov/pubmed/25983043







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2014

Bachanova V, Burns LJ, Wang T, Carreras J, Gale RP, Wiernik PH, Ballen KK, Wirk B, Munker R, Rizzieri DA, Chen Y-B, Gibson J, Akpek G, Costa LJ, Kamble RT, Aljurf MD, Hsu JW, Cairo MS, Schouten HC, Bacher U, Savani BN, Wingard JR, Lazarus HM, Laport GG, Montoto S, Maloney DG, Smith SM, Brunstein C, Saber W. Alternative donors extend transplantation for patients with lymphoma who lack an HLA matched donor. Bone Marrow Transplantation. 2015 Feb 7; 50(2):197-203. doi:10.1038/bmt.2014.259. Epub 2014 Nov 17. PMC4336786. ncbi.nlm.nih.gov/ pubmed/25402415

Lechowicz MJ, Lazarus HM, Carreras J, Laport GG, Cutler CS, Wiernik PH, Hale GA, Maharaj D, Gale RP, Rowlings PA, Freytes CO, Miller AM, Vose JM, Maziarz RT, Montoto S, Maloney DG, Hari PN. Allogeneic hematopoietic cell transplantation for mycosis fungoides and Sézary syndrome. Bone Marrow Transplantation. 2014 Nov 1; 49(11):1360-1365. doi:10.1038/bmt. 2014.161. Epub 2014 Jul 28. PMC4221526. ncbi.nlm.nih.gov/pubmed/25068422

Hamadani M, Hari PN, Zhang Y, Carreras J, Akpek G, Aljurf MD, Ayala E, Bachanova V, Chen AI, Chen Y-B, Costa LJ, Fenske TS, Freytes CO, Ganguly S, Hertzberg MS, Holmberg LA, Inwards DJ, Kamble RT, Kanfer EJ, Lazarus HM, Marks DI, Nishihori T, Olsson R, Reddy NM, Rizzieri DA, Savani BN, Solh M, Vose JM, Wirk B, Maloney DG, Smith SM, Montoto S, Saber W. Early failure of frontline rituximab-containing chemo-immunotherapy in diffuse large B cell lymphoma does not predict futility of autologous hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1729-1736. doi:10.1016/j.bbmt.2014.06.036. Epub 2014 Jul 5. PMC4194275. ncbi.nlm.nih.gov/pubmed/25008330


LY17-01b Title: Clinical cutcomes of patients age ≥65 undergoing allogeneic hematopoietic cell

transplant for non-Hodgkin lymphoma PI: Nirav Shah (Medical College of Wisconsin) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) LY17-02a Title: Allografts following reduced intensity conditioning for Hodgkin's lymphoma PI: Nilanjan Ghosh (Levine Cancer Institute) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LY17-02b Title: Allografts following reduced intensity conditioning for non-Hodgkin disease PI: Sairah Ahmed (M.D. Anderson Cancer Center) Status: Manuscript Preparation (as of July 1, 2019)







Page | 85

LY17-02c Title: Conditioning regimen in allografts for diffuse large B cell lymphoma PI: Mehdi Hamadani (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LY17-02d Title: 2 versus 4 centigray fludarabine / total body irradiation in allografts for non-

Hodgkin lymphoma PI: Mehdi Hamadani (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)


LY18-01 Title: Outcomes in B cell non-Hodgkin’s lymphoma patients who underwent autologous

stem cell transplantation following rituximab containing conditioning regimens PIs: Deepa Jagadeesh (Cleveland Clinic Foundation) Navneet Majhail (Cleveland Clinic Foundation) Brian Hill (Cleveland Clinic Foundation) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LY18-02 Title: Effect of time to relapse on overall survival in mantle cell lymphoma patients

following frontline autologous stem cell transplant PIs: Peter Riedell (University of Chicago Medicine) Sonali Smith (University of Chicago Medicine) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) LY18-03 Title: Transplantation for CLL undergoing Richter’s transformation arising in the

setting of indolent lymphoma PI: Alex Herrera (City of Hope) Status: Data File Preparation (as of July 1, 2019)



Page | 86

LY19-01 Title: Post-transplant cyclophosphamide-based haploidentical transplantation versus

matched sibling or well matched unrleated donor transplantation for peripheral T-cell lymphoma: A CIBMTR Lymphoma Working Committee and EBMT Lymphoma Working Party analysis

PIs: Peter Dreger (Universitaetsklinikum Heidelberg) Mehdi Hamadani (Medical College of Wisconsin) Status: Protocol Pending (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) * Collaborative study with EBMT LY19-02 Title: Determining the optimal conditioning regimen for patients with primary central

nervous system lymphoma undergoing autologous hematopoietic cell tranplantation

PIs: Michael Scordo (Memorial Sloan Kettering Cancer Center) Craig Sauter (Memorial Sloan Kettering Cancer Center) Antonio Jimenez (University of Miami) Status: Draft Protocol Received (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 13.0 NON-MALIGNANT DISEASES WORKING COMMITTEE

Page | 87

13.0 NON-MALIGNANT DISEASES WORKING COMMITTEE

13.1 Leadership

Chair: Vikram Mathews, MD, Christian Medical College Hospital Email: [email protected] Chair: Christopher Dvorak, MD, University of California San Francisco Medical

Center Email: [email protected] Chair: Andrew Gennery, MD, Newcastle General Hospital and The Royal Victoria

Infirmary Email: [email protected] Chair: George Georges, MD, Fred Hutchinson Cancer Research Center Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Soyoung Kim, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Kyle Hebert, MS, CIBMTR Milwaukee Email: [email protected]


2018

Rice C, Eikema DJ, Marsh JCW, Knol C, Hebert K, Putter H, Peterson E, Deeg HJ, Halkes S, Pidala J, Anderlini P, Tischer J, Kroger N, McDonald A, Antin JH, Schaap NP, Hallek M, Einsele H, Mathews V, Kapoor N, Boelens JJ, Mufti GJ, Potter V, Pefault de la Tour R, Eapen M, Dufour C. Allogeneic hematopoietic cell transplantation in patients aged 50 years or older with severe aplastic anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Mar 1; 25(3):488-495. doi:10.1016/j.bbmt. 2018.08.029. Epub 2018 Sep 5. PMC6401340. ncbi.nlm.nih.gov/pubmed/30194027

Marsh RA, Hebert KM, Keesler D, Boelens JJ, Dvorak CC, Eckrich MJ, Kapoor N, Parikh S, Eapen M. Practice pattern changes and improvements in hematopoietic cell transplantation for primary immunodeficiencies. Journal of Allergy and Clinical Immunology. 2018 Dec 1; 142(6):2004-2007. doi:10.1016/j.jaci.2018.08.010. Epub 2018 Aug 28. PMC6289686. ncbi.nlm.nih.gov/pubmed/30170121











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2017

Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC. Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: A PIDTC natural history study. Blood. 2017 Dec 21; 130(25):2718-2727. doi:10.1182/blood-2017-05-781849. Epub 2017 Oct 11. PMC5746165. ncbi.nlm.nih.gov/pubmed/29021228

Abraham A, Hsieh M, Eapen M, Fitzhugh C, Carreras J, Keesler D, Guilcher G, Kamani N, Walters MC, Boelens JJ, Tisdale J, Shenoy S. Relationship between mixed donor-recipient chimerism and disease recurrence after hematopoietic cell transplantation for sickle cell disease. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Dec 1; 23(12):2178-2183. doi:10.1016/j.bbmt.2017.08.038. Epub 2017 Sep 4. PMC5782809. ncbi.nlm.nih.gov/pubmed/28882446

Slack J, Albert MH, Balashov D, Belohradsky BH, Bertaina A, Bleesing J, Booth C, Büchner J, Buckley RH, Ouachée-Chardin M, Deripapa E, Drabko K, Eapen M, Feuchtinger T, Finocchi A, Gaspar HB, Ghosh S, Gillio A, Gonzalez-Granado LI, Grunebaum E, Güngör T, Heilmann C, Heminen M, Higuchi K, Imai K, Kalwak K, Kanazawa N, Karasu G, Kucuk ZY, Laberko A, Lange A, Mahlaoui N, Meisel R, Moshous D, Muramatsu H, Parikh S, Pasic S, Schmid I, Schuetz C, Schulz A, Schultz KR, Shaw PJ, Slatter MA, Sykora K-W, Tamura S, Taskinen M, Wawer A, Wolska-Kuśnierz B, Cowan MJ, Fischer A, Gennery AR. Outcome of hematopoietic cell transplantation for DNA-double strand breakage repair disorders. 2018 Jan 1; 141(1):322-328.e10. doi:10.1016/ j.jaci.2017.02.036. Epub 2017 Apr 7. PMC5632132. ncbi.nlm.nih.gov/pubmed/28392333

Kekre N, Zhang Y, Zhang M-J, Carreras J, Ahmed P, Anderlini P, Atta EH, Ayas M, Boelens JJ, Bonfim C, Deeg HJ, Kapoor N, Lee J-W, Nakamura R, Pulsipher MA, Eapen M, Antin JH. Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia. Haematologica. 2017 Jul 1; 102(7):1291-1298. doi:10.3324/haematol.2017. 164459. Epub 2017 Mar 24. PMC5566045. ncbi.nlm.nih.gov/pubmed/28392333

2016

Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle J-H, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017 Mar 16; 129(11):1548-1556. doi:10.1182/blood-2016-10-745711. Epub 2016 Dec 13. PMC5356458. ncbi.nlm.nih.gov/ pubmed/27965196








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Gadalla SM, Wang T, Dagnall C, Haagenson M, Spellman SR, Hicks B, Jones K, Katki HA, Lee SJ, Savage SA. Effect of recipient age and stem cell source on the association between donor telomere length and survival after allogeneic unrelated hematopoietic cell transplantation for severe aplastic anemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Dec 1; 22(12):2276-2282. doi:10.1016/j.bbmt.2016.09.012. Epub 2016 Sep 15. PMC5116252. ncbi.nlm.nih.gov/pubmed/27641680

2015

Ayas M, Eapen M, Le-Rademacher J, Carreras J, Abdel-Azim H, Alter BP, Anderlini P, Battiwalla M, Bierings M, Buchbinder DK, Bonfim C, Camitta BM, Fasth AL, Gale RP, Lee MA, Lund TC, Myers KC, Olsson RF, Page KM, Prestidge TD, Radhi M, Shah AJ, Schultz KR, Wirk B, Wagner JE, Deeg HJ. Second allogeneic hematopoietic cell transplantation for patients with Fanconi anemia and bone marrow failure. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):1790-1795. doi:10.1016/j.bbmt.2015.06.012. Epub 2015 Jun 23. PMC4568139. ncbi.nlm.nih.gov/pubmed/ 26116087

Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, Al-Seraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Diaz Perez MA, Veys PA, Eapen M. Hematopoietic stem cell transplantation for infantile osteopetrosis. Blood. 2015 Jul 9; 126(2):270-276. doi:10.1182/blood-2015-01-625541. Epub 2015 May 26. PMC4497967. ncbi.nlm.nih.gov/pubmed/26012570

Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M. Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning. British Journal of Haematology. 2015 Jun 1; 169(5):711-718. doi:10.1111/bjh.13347. Epub 2015 Mar 27. PMC4433436. ncbi.nlm.nih.gov/pubmed/ 25817915

2014

Kim S-Y, Le Rademacher J, Antin JH, Anderlini P, Ayas M, Battiwalla M, Carreras J, Kurtzberg J, Nakamura R, Eapen M, Deeg HJ. Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: Efficacy of hematopoietic stem cell transplantation. Haematologica. 2014 Dec 1; 99(12):1868-1875. doi:10.3324/haematol. 2014.108977. Epub 2014 Aug 8. PMC4258748. ncbi.nlm.nih.gov/pubmed/25107891

Lund TC, Cathey SS, Miller WP, Eapen M, Andreansky M, Dvorak CC, Davis JH, Dalal JD, Devine SM, Eames GM, Ferguson WS, Giller RH, He W, Kurtzberg J, Krance R, Katsanis E, Lewis VA, Sahdev I, Orchard PJ. Outcomes after hematopoietic stem cell transplantation for children with I-cell disease. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1847-1851. doi:10.1016/j.bbmt.2014. 06.019. Epub 2014 Jul 10. PMC4194244. ncbi.nlm.nih.gov/pubmed/25016194










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AA13-02 Title: Malignancies in patients with fanconi anemia PI: John Wagner (University of Minnesota Blood and Marrow Transplant Program) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with the National Cancer Institute AC18-02 Title: Prospective cohort study of recipients of autologous hematopoietic cell transplant

for systemic sclerosis PIs: George Georges (Fred Hutchinson Cancer Research Center) Keith Sullivan (Duke University Medical Center) Status: Protocol Development (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) ID13-01 Title: Haematopoietic stem cell transplants for congenital neutropenia / Kostmann

agranulocytosis PIs: Cornelia Zeidler (Medizinische Hochschule Hannover) Steven Keogh (The Children's Hospital at Westmead) James Connelly (Vanderbilt University Medical Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Severe Chronic Neutropenia International Registry NM14-02 Title: Outcomes of allogeneic hematopoietic cell transplant in patients with Shwachman

Diamond syndrome PI: Kasiani Myers (Cincinnati Children's Hospital Medical Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) NM15-01 Title: Outcome of allogeneic hematopoietic cell transplant in erythropoietic porphyria PIs: Ayman Saad (Ohio State Medical Center, James Cancer Center) Hisham Abdel-Azim (Children's Hospital of Los Angeles) Joseph Bloomer (University of Alabama at Birmingham) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with EBMT


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NM16-03 Title: Results of transplants from genetically-identical twin donors in persons with

aplastic anemia PI: Robert Peter Gale (LifebankUSA-Celularity) Status: Analysis (as of July 1, 2019)

Submitted (expected by June 30, 2020) NM16-04 Title: The effect of conditioning regimen on clinical outcomes of allogeneic

hematopoietic cell transplantation in severe aplastic anemia PIs: Nelli Bejanyan (H. Lee Moffitt Cancer Center and Research Institute) Natasha Kekre (The Ottawa Hospital Blood & Marrow Transplant Program) Daniel Weisdorf (University of Minnesota Blood and Marrow Transplant Program) Joseph Antin (Dana Farber Cancer Institute) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) NM17-01 Title: Late effects after hematopoietic stem cell transplantation in patients with

hemophagocytic lymphohistiocytosis PIs: AnnaCarin Horne (Karolinska Institutet) K Scott Baker (Fred Hutchinson Cancer Center) Karin Beutel (Histiocyte Society) Status: Protocol Development (as of July 1, 2019)

Analysis (expected by June 30, 2020) * Collaborative study with EBMT NM17-03b Title: Risk factor scoring for allogeneic hematopoietic stem cell transplantation for

sickle cell disease PI: Ruta Brazauskas (Medical College of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) NM18-01 Title: Impact of choice of serotherapy in pediatric stem cell transplantation for non-

malignant disease PIs: Anand Prakash (Hospital for Sick Children, Toronto) Donna Wall (Hospital for Sick Children) Kristjian Paulson (CancerCare Manitoba/University of Manitoba) Status: Protocol Development (as of July 1, 2019)



Page | 92

NM19-01 Title: Conditional and cause-specific mortality of patients with severe aplastic anemia

surviving at least one year after alloHCT or immunosuppressive therapy PIs: Ryotaro Nakamura (City of Hope) F. Lennie Wong (City of Hope) Saro Armenian (City of Hope) Status: Protocol Pending (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) * Collaborative study with National Heart, Lung, and Blood Institute NM19-02 Title: Impact of reduced intensity conditioning on allogeneic HCT outcomes for HLH PI: Rebecca Marsh (Cincinnati Children's Hospital Medical Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) NM19-03 Title: Hematopoietic stem cell transplantation for congenital amegakaryocytic

thrombocytopenia PIs: Farid Boulad (Memorial Sloan Kettering Cancer Center) Maria Cancio (Memorial Sloan Kettering Cancer Center) Jaap Jan Boelens (Memorial Sloan Kettering Cancer Center) Status: Draft Protocol Received (as of July 1, 2019)

Data File Preparation (expected by June 30, 2020) SC17-09/17-CMS-SCD Title: Prospective assessment of allogeneic hematopoietic cell transplantation in

adolescents and young adults with severe sickle cell disease PI: Mary Eapen (Medical College of Wisconsin) Status: Data Collection/Data File Preparation (as of July 1, 2019)

Data Collection/Data File Preparation (expected by June 30, 2020)

2019 CIBMTR Scientific Working Committee Research Portfolio 14.0 PEDIATRIC CANCER WORKING COMMITTEE

Page | 93

14.0 PEDIATRIC CANCER WORKING COMMITTEE

14.1 Leadership

Chair: Angela Smith, MD, MS, University of Minnesota Medical Center, Fairview Email: [email protected] Chair: Gregory Yanik, MD, The University of Michigan Email: [email protected] Chair: Muna Qayed, Children’s Healthcare of Atlanta at Egleston Email: [email protected] Scientific Director: Mary Eapen, MD, MS, CIBMTR Milwaukee Email: [email protected] Statistical Director: Kwang Woo Ahn, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Kyle Hebert, MS, CIBMTR Milwaukee Email: [email protected]


2018

Lund TC, Ahn KW, Tecca HR, Hilgers MV, Abdel-Azim H, Abraham A, Diaz MA, Badawy SM, Broglie L, Brown V, Dvorak CC, Gonzalez-Vicent M, Hashem H, Hayashi RJ, Jacobsohn DA, Kent MW, Li C-K, Margossian SP, Martin PL, Mehta P, Myers K, Olsson R, Page K, Pulsipher MA, Shaw PJ, Smith AR, Triplett BM, Verneris MR, Eapen M. Outcomes after second hematopoietic cell transplantation in children and young adults with relapsed acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Feb 1; 25(2):301-306. doi:10.1016/j.bbmt.2018.09.016. Epub 2018 Sep 19. PMC6339844. ncbi.nlm.nih.gov/pubmed/30244103

2017

Malogolowkin MH, Hemmer MT, Le-Rademacher J, Hale GA, Mehta PA, Smith AR, Kitko C, Abraham A, Abdel-Azim H, Dandoy C, Angel Diaz M, Gale RP, Guilcher G, Hayashi R, Jodele S, Kasow KA, MacMillian ML, Thakar M, Wirk BM, Woolfrey A, Thiel EL. Outcomes following autologous hematopoietic stem cell transplant for patients with relapsed Wilms' tumor: A CIBMTR retrospective analysis. Bone Marrow Transplantation. 2017 Nov 1; 52(11):1549-1555. doi:10.1038/bmt.2017.178. Epub 2017 Sep 4. PMC5665725. ncbi.nlm.nih.gov/pubmed/ 28869618











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Bitan M, Ahn KW, Millard HR, Pulsipher MA, Abdel-Azim H, Auletta JJ, Brown V, Chan KW, Diaz MA, Dietz A, Vincent MG, Guilcher G, Hale GA, Hayashi RJ, Keating A, Mehta P, Myers K, Page K, Prestidge T, Shah NN, Smith AR, Woolfrey A, Thiel E, Davies SM, Eapen M. Personalized prognostic risk score for long-term survival for children with acute leukemia after allogeneic transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Sep 1; 23(9):1523-1530. doi:10.1016/j.bbmt.2017.05.011. Epub 2017 May 17. PMC5683075. ncbi.nlm.nih.gov/pubmed/ 28527984

Khandelwal P, Millard HR, Thiel E, Abdel-Azim H, Abraham AA, Auletta JJ, Boulad F, Brown VI, Camitta BM, Chan KW, Chaudhury S, Cowan MJ, Angel-Diaz M, Gadalla SM, Gale RP, Hale G, Kasow KA, Keating AK, Kitko CL, MacMillan ML, Olsson RF, Page KM, Seber A, Smith AR, Warwick AB, Wirk B, Mehta PA. Hematopoietic stem cell transplant activity in pediatric cancer between 2008 and 2014 in the United States: A CIBMTR report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Aug 1; 23(8):1342-1349. doi:10.1016/j.bbmt.2017.04.018. Epub 2017 Apr 24. PMC5669065. ncbi.nlm.nih.gov/pubmed/28450183

2015

Burke MJ, Verneris MR, Le Rademacher J, He W, Abdel-Azim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL. Transplant outcomes for children with T cell acute lymphoblastic leukemia in second remission: A report from the Center for International Blood and Marrow Transplant Research. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):2154-2159. doi:10.1016/j.bbmt.2015.08.023. Epub 2015 Aug 29. PMC4654112. ncbi.nlm.nih.gov/pubmed/26327632

Mehta PA, Zhang M-J, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel-Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM. Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1273-1277. doi:10.1016/j.bbmt.2015.04.008. Epub 2015 Apr 10. PMC4465998. ncbi.nlm.nih.gov/pubmed/25865650


PC18-01 Title: Comparison of TBI vs non-TBI based regimens for pediatric AML in the modern

era PI: Christopher Dandoy (Cincinnati Children's Hospital Medical Center) Status: Manuscript Preparation (as of July 1, 2019)








Page | 95

PC19-01 Title: Variation of the disease risk index in children undergoing alloHCT PIs: M Qayed (Children's Healthcare of Atlanta at Egleston) C Kitko (Vanderbilt University Medical Center) Status: Protocol Pending (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) PC19-02 Title: Does mixed peripheral blood T cell chimerism predict relapse? PIs: S Prockop (Memorial Sloan Kettering Cancer Center) J Boelens (Memorial Sloan Kettering Cancer Center) K Peggs (University College London) Status: Protocol Pending (as of July 1, 2019)

Analysis (expected by June 30, 2020) PC19-03 Title: The impact of pre-transplant extramedullary disease on the outcome of allogeneic

hematopoietic cell transplantation for acute myeloid leukemia in children- A combined CIBMTR and EBMT analysis

PIs: H Rangarajan (Nationwide Children's Hospital) P Satwani (NYPH/ Columbia University Medical Center) K Rao (University of North Carolina Hospitals) D Chellapandian (Johns Hopkins All Children's Hospital) B Savani (Vanderbilt University Medical Center) Juliana Silva (Great Ormond Street Hospital for Children NHS Foundation Trust) Status: Data File Preparation (as of July 1, 2019)

Analysis (expected by June 30, 2020) * Collaborative study with EBMT

2019 CIBMTR Scientific Working Committee Research Portfolio 15.0 PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE

Page | 96

15.0 PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE

15.1 Leadership

Chair: Shaji Kumar, Mayo Clinic Rochester Email: [email protected] Chair: Nina Shah, MD, University of California San Francisco Medical Center Email: [email protected] Chair: Muzaffar Qazilbash, MD, MD Anderson Cancer Center Email: [email protected] Scientific Director: Parameswaran Hari, MD, MS, CIBMTR Milwaukee Email: [email protected] Asst Sci Director: Anita D’Souza, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Raphael Fraser, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Noel Estrada-Merly, MS, CIBMTR Milwaukee Email: [email protected]


2019

Kilari D, D'Souza A, Fraser R, Qayed M, Davila O, Agrawal V, Diaz MA, Chhabra S, Cerny J, Copelan E, Farhadfar N, Freytes CO, Gale RP, Ganguly S, Hildebrandt GC, Holmberg L, Kamble RT, Kapoor N, Lazarus H, Lee C, Murthy HS, Naik S, Nishihori T, Saad A, Savani BN, Seo S, Warwick A, Wirk B, Yared JA, Nieto Y, Hari P. Autologous transplantation for male germ cell tumors: improved outcomes over 3 decades. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Jun 1; 25(6):1099-1106. doi:10.1016/j.bbmt.2019.02.015. Epub 2019 Feb 20. PMC6559839. ncbi.nlm.nih.gov/pubmed/ 30794931

2018

Gopalakrishnan S, D'Souza A, Scott E, Fraser R, Davila O, Shah N, Gale RP, Kamble R, Diaz MA, Lazarus HM, Savani BN, Hildebrandt GC, Solh M, Freytes CO, Lee C, Kyle RA, Usmani SZ, Ganguly S, Assal A, Berdeja J, Kanate AS, Dhakal B, Meehan K, Kindwall-Keller T, Saad A, Locke F, Seo S, Nishihori T, Gergis U, Gasparetto C, Mark T, Nieto Y, Kumar S, Hari P. Revised-International Staging System (R-ISS) is predictive and prognostic for early relapse (<24 months) after autologous transplantation for newly diagnosed multiple myeloma (MM). Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.12.141. Epub 2018 Dec 20. 30579965. ncbi.nlm.nih.gov/pubmed/30579965











Page | 97

Sahebi F, Garderet L, Kanate AS, Eikema DJ, Knelange NS, Alvelo OFD, Koc Y, Blaise D, Bashir Q, Moraleda JM, Dreger P, Sanchez JF, Ciurea S, Schouten H, Shah NN, Verbeek M, Rösler W, Diez-Martin JL, Schoenland S, D'Souza A, Kröger N, Hari P. Outcomes of haploidentical transplantation in patients with relapsed multiple myeloma: An EBMT / CIBMTR report. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Feb 1; 25(2):335-342. doi:10.1016/j.bbmt.2018.09.018. Epub 2018 Sep 20. PMC6339830. ncbi.nlm.nih.gov/pubmed/30243581

Scott EC, Hari P, Kumar S, Fraser R, Davila O, Shah N, Gale RP, Diaz MA, Agrawal V, Cornell RF, Ganguly S, Akpek G, Freytes C, Hashmi S, Malek E, Kamble RT, Lazarus H, Solh M, Usmani SZ, Kanate AS, Saad A, Chhabra S, Gergis U, Cerny J, Kyle RA, Lee C, Kindwall-Keller T, Assal A, Hildebrandt GC, Holmberg L, Maziarz RT, Nishihori T, Seo S, Kumar S, Mark T, D'Souza A. Staging systems for newly diagnosed myeloma patients undergoing autologous hematopoietic cell transplantation: The Revised International Staging System shows the most differentiation between groups. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Dec 1; 24(12):2443-2449. doi:10.1016/j.bbmt. 2018.08.013. Epub 2018 Aug 22. PMC6293469. ncbi.nlm.nih.gov/pubmed/30142419

2017

Kumar SK, Dispenzieri A, Fraser R, Mingwei F, Akpek G, Cornell R, Kharfan-Dabaja M, Freytes C, Hashmi S, Hildebrandt G, Holmberg L, Kyle R, Lazarus H, Lee C, Mikhael J, Nishihori T, Tay J, Usmani S, Vesole D, Vij R, Wirk B, Krishnan A, Gasparetto C, Mark T, Nieto Y, Hari P, D'Souza A. Early relapse after autologous hematopoietic cell transplantation remains a poor prognostic factor in multiple myeloma but outcomes have improved over time. Leukemia. 2018 Apr 1; 32(4):986-995. doi:10.1038/leu.2017.331. Epub 2017 Nov 16. PMC5871538. ncbi.nlm.nih.gov/pubmed/29263438

Htut M, D'Souza A, Krishnan A, Bruno B, Zhang M-J, Fei M, Diaz MA, Copelan E, Ganguly S, Hamadani M, Kharfan-Dabaja M, Lazarus H, Lee C, Meehan K, Nishihori T, Saad A, Seo S, Ramanathan M, Usmani SZ, Gasparetto C, Mark TM, Nieto Y, Hari P. Autologous/allogeneic hematopoietic cell transplantation versus tandem autologous transplantation for multiple myeloma: Comparison of long-term postrelapse survival. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2018 Mar 1; 24(3):478-485. doi:10.1016/j.bbmt.2017.10.024. Epub 2017 Oct 24. PMC5826888. ncbi.nlm.nih.gov/pubmed/29079457

Mahindra A, Hari P, Fraser R, Fei M, Huang J, Berdeja J, Callander N, Costa L, Diaz MA, Freytes C, Gale RP, Girnius S, Holmberg L, Kharfan-Dabaja M, Kumar S, Kyle R, Lazarus H, Lee C, Maiolino A, Moreb J, Nishihori T, Pawarode A, Saad A, Savani BN, Schriber J, William B, Wirk BM, Krishnan A, Nieto Y, D'Souza A. Autologous hematopoietic cell transplantation for multiple myeloma patients with renal insufficiency: A Center for International Blood and Marrow Transplant Research analysis. Bone Marrow Transplantation. 2017 Dec 1; 52(12):1616-1622. doi:10.1038/ bmt.2017.198. Epub 2017 Sep 18. PMC5859934. ncbi.nlm.nih.gov/pubmed/28920949







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D'Souza A, Zhang M-J, Huang J, Fei M, Pasquini M, Hamadani M, Hari P. Trends in pre- and post-transplant therapies with first autologous hematopoietic cell transplantation among patients with multiple myeloma in the United States, 2004-2014. Leukemia. 2017 Sep 1; 31(9):1998-2000. doi:10.1038/leu.2017.185. Epub 2017 Jun 13. PMC5587375. ncbi.nlm.nih.gov/pubmed/28663578

Schriber JR, Hari PN, Ahn KW, Fei M, Costa LJ, Kharfan-Dabaja MA, Angel-Diaz M, Gale RP, Ganguly S, Girnius SK, Hashmi S, Pawarode A, Vesole DH, Wiernik PH, Wirk BM, Marks DI, Nishihori T, Olsson RF, Usmani S, Mark TM, Nieto YL, D'Souza A. Hispanics have the lowest stem cell transplant utilization rate for autologous hematopoietic cell transplantation for multiple myeloma in the United States: A CIBMTR report. Cancer. 2017 Aug 15; 123(16):3141-3149. doi:10.1002/cncr.30747. Epub 2017 May 4. PMC5544566. ncbi.nlm.nih.gov/pubmed/28472539

Cheng YC, Shi Y, Zhang M-J, Brazauskas R, Hemmer MT, Bishop MR, Nieto Y, Stadtmauer E, Ayashi L, Gale RP, Lazarus H, Holmberg L, Lill M, Olsson RF, Wirk BM, Arora M, Hari P, Ueno N. Long-term outcome of inflammatory breast cancer compared to non-inflammatory breast cancer in the setting of high-dose chemotherapy with autologous hematopoietic cell transplantation. Journal of Cancer. 8(6):1009-1017. doi:10.7150/jca.16870. Epub 2017 Mar 25. PMC5436253. ncbi.nlm.nih.gov/pubmed/28529613

Garderet L, D'Souza A, Jacobs P, van Biezen A, Schönland S, Kroeger N, Morris C, Hari P. Response assessment in myeloma: Practical manual on consistent reporting in an era of dramatic therapeutic advances. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jul 1; 23(7):1193-1202. doi:10.1016/j.bbmt.2017.03.009. Epub 2017 Mar 8. ncbi.nlm.nih.gov/pubmed/28285082

2016

Cornell RF, D'Souza A, Kassim AA, Costa LJ, Innis-Shelton RD, Zhang M-J, Huang J, Abidi M, Aiello J, Akpek G, Bashey A, Bashir Q, Cerny J, Comenzo R, Diaz MA, Freytes C, Gale RP, Ganguly S, Hamadani M, Hashmi S, Holmberg L, Hossain N, Kamble RT, Kharfan-Dabaja M, Kindwall-Keller T, Kyle R, Kumar S, Lazarus H, Lee C, Maiolino A, Marks DI, Meehan K, Mikhael J, Nath R, Nishihori T, Olsson RF, Ramanathan M, Saad A, Seo S, Usmani S, Vesole D, Vij R, Vogl D, Wirk BM, Yared J, Krishnan A, Mark T, Nieto Y, Hari P. Maintenance versus induction therapy choice on outcomes after autologous transplantation for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Feb 1; 23(2):269-277. doi:10.1016/j.bbmt.2016.11.011. Epub 2016 Nov 15. PMC5346183. ncbi.nlm.nih.gov/pubmed/27864161

Cornell RF, Bachanova V, D'Souza A, Woo-Ahn K, Martens M, Huang J, Al-Homsi AS, Chhabra S, Copelan E, Diaz M-A, Freytes CO, Gale RP, Ganguly S, Hamadani M, Hildebrandt G, Kamble RT, Kharfan-Dabaja M, Kindwall-Keller T, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Saad A, Usmani S, Vesole DH, Yared J, Mark T, Nieto Y, Hari P. Allogeneic transplantation for relapsed Waldenström macroglobulinemia and lymphoplasmacytic lymphoma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Jan 1; 23(1):60-66. doi:10.1016/j.bbmt.2016.10.010. Epub 2016 Oct 24. PMC5182098. ncbi.nlm.nih.gov/pubmed/27789362








Page | 99

Scott EC, Hari P, Sharma M, Le-Rademacher J, Huang J, Vogl D, Abidi M, Beitinjaneh A, Fung H, Ganguly S, Hildebrandt G, Holmberg L, Kalaycio M, Kumar S, Kyle R, Lazarus H, Lee C, Maziarz RT, Meehan K, Mikhael J, Nishihori T, Ramanathan M, Usmani S, Tay J, Vesole D, Wirk B, Yared J, Savani BN, Gasparetto C, Krishnan A, Mark T, Nieto Y, D'Souza A. Post-transplant outcomes in high-risk compared with non-high-risk multiple myeloma: A CIBMTR analysis. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Oct 1; 22(10):1893-1899. doi:10.1016/j.bbmt.2016.07.007. Epub 2016 Aug 2. PMC5090978. ncbi.nlm.nih.gov/pubmed/27496215

Beksac M, Gragert L, Fingerson S, Maiers M, Zhang M-J, Albrecht M, Zhong X, Cozen W, Dispenzieri A, Lonial S, Hari P. HLA polymorphism and risk of multiple myeloma. Leukemia. 2016 Nov 1; 30(11):2260-2264. doi:10.1038/leu.2016.199. Epub 2016 Jul 27. PMC5134914. ncbi.nlm.nih.gov/pubmed/27461064

Rodriguez TE, Hari P, Stiff PJ, Smith SE, Sterrenberg D, Vesole DH. Busulfan, melphalan, and bortezomib versus high-dose melphalan as a conditioning regimen for autologous hematopoietic stem cell transplantation in multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug; 22(8):1391-6. doi: 10.1016/j.bbmt.2016.03.021. Epub 2016 May 7. ncbi.nlm.nih.gov/ pubmed/27164062

2015

D'Souza A, Huang J, Hari P. New light chain amyloid response criteria help risk stratification of patients by day 100 after autologous hematopoietic cell transplant. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Apr 1; 22(4):768-770. doi:10.1016/j.bbmt.2015.12.021. Epub 2015 Dec 29. PMC4801777. ncbi.nlm.nih.gov/pubmed/26743343

D'Souza A, Dispenzieri A, Wirk B, Zhang M-J, Huang J, Gertz MA, Kyle RA, Kumar S, Comenzo RL, Gale RP, Lazarus HM, Savani BN, Cornell RF, Weiss BM, Vogl DT, Freytes CO, Scott EC, Landau HJ, Moreb JS, Costa LJ, Ramanathan M, Callander NS, Kamble RT, Olsson RF, Ganguly S, Nishihori T, Kindwall-Keller TL, Wood WA, Mark TM, Hari P. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: A Center for International Blood and Marrow Transplant Research study. Journal of Clinical Oncology. 2015 Nov 10; 33(32):3741-3749. doi:10.1200/JCO.2015.62.4015. Epub 2015 Sep 14. PMC4737858. ncbi.nlm.nih.gov/pubmed/26371138

Uy GL, Costa LJ, Hari PN, Zhang M-J, Huang J-X, Anderson KC, Bredeson CN, Callander NS, Cornell RF, Perez MAD, Dispenzieri A, Freytes CO, Gale RP, Garfall A, Gertz MA, Gibson J, Hamadani M, Lazarus HM, Kalaycio ME, Kamble RT, Kharfan-Dabaja MA, Krishnan AY, Kumar SK, Kyle RA, Landau HJ, Lee CH, Maiolino A, Marks DI, Mark TM, Munker R, Nishihori T, Olsson RF, Ramanathan M, Rodriguez TE, Saad AA, Savani BN, Schiller GJ, Schouten HC, Schriber JR, Scott E, Seo S, Sharma M, Ganguly S, Stadtmauer EA, Tay J, To LB, Vesole DH, Vogl DT, Wagner JL, Wirk B, Wood WA, D'Souza A. Contribution of chemotherapy mobilization to disease control in multiple myeloma treated with autologous hematopoietic cell transplantation. Bone Marrow Transplantation. 2015 Dec 1; 50(12):1513-1518. doi:10.1038/bmt.2015.190. Epub 2015 Aug 24. PMC4548821. ncbi.nlm.nih.gov/pubmed/26301967



http://www.ncbi.nlm.nih.gov/pubmed/?term=Rodriguez%20TE%5BAuthor%5D&cauthor=true&cauthor_uid=27164062

http://www.ncbi.nlm.nih.gov/pubmed/?term=Hari%20P%5BAuthor%5D&cauthor=true&cauthor_uid=27164062

http://www.ncbi.nlm.nih.gov/pubmed/?term=Stiff%20PJ%5BAuthor%5D&cauthor=true&cauthor_uid=27164062

http://www.ncbi.nlm.nih.gov/pubmed/?term=Smith%20SE%5BAuthor%5D&cauthor=true&cauthor_uid=27164062

http://www.ncbi.nlm.nih.gov/pubmed/?term=Sterrenberg%20D%5BAuthor%5D&cauthor=true&cauthor_uid=27164062

http://www.ncbi.nlm.nih.gov/pubmed/?term=Vesole%20DH%5BAuthor%5D&cauthor=true&cauthor_uid=27164062







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2014

Mahindra A, Raval G, Mehta P, Brazauskas R, Zhang M-J, Zhong X, Bird JM, Freytes CO, Hale GA, Herzig R, Holmberg LA, Kamble RT, Kumar S, Lazarus HM, Majhail NS, Marks DI, Moreb JS, Olsson R, Saber W, Savani BN, Schiller GJ, Tay J, Vogl DT, Waller EK, Wiernik PH, Wirk B, Lonial S, Krishnan AY, Dispenzieri A, Brandenburg NA, Gale RP, Hari PN. New cancers after autotransplantations for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):738-745. doi:10.1016/j.bbmt.2014.12.028. Epub 2014 Dec 31. PMC4359647. ncbi.nlm.nih.gov/ pubmed/25555448

Costa LJ, Huang J-X, Hari PN. Disparities in utilization of autologous hematopoietic cell transplantation for treatment of multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):701-706. doi:10.1016/j.bbmt.2014.12.024. Epub 2014 Dec 30. PMC4361014. ncbi.nlm.nih.gov/pubmed/25555447

Vij R, Kumar S, Zhang M-J, Zhong X, Huang J, Dispenzieri A, Abidi MH, Bird JM, Freytes CO, Gale RP, Kindwall-Keller TL, Kyle RA, Landsburg DJ, Lazarus HM, Munker R, Roy V, Sharma M, Vogl DT, Wirk B, Hari PN. Impact of pretransplant therapy and depth of disease response before autologous transplantation for multiple myeloma. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Feb 1; 21(2):335-341. doi:10.1016/j.bbmt.2014.10.023. Epub 2014 Nov 1. PMC4297511. ncbi.nlm.nih.gov/pubmed/25445028

Sharma M, Zhang M-J, Zhong X, Abidi MH, Akpek G, Bacher U, Callander NS, Dispenzieri A, Freytes CO, Fung HC, Gale RP, Gasparetto C, Gibson J, Holmberg LA, Kindwall-Keller TL, Klumpp TR, Krishnan AY, Landau HJ, Lazarus HM, Lonial S, Maiolino A, Marks DI, Mehta P, Mikhael Med JR, Nishihori T, Olsson R, Ramanathan M, Roy V, Savani BN, Schouten HC, Scott E, Tay J, To LB, Vesole DH, Vogl DT, Hari P. Older patients with myeloma derive similar benefit from autologous transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2014 Nov 1; 20(11):1796-1803. doi:10.1016/j.bbmt. 2014.07.013. Epub 2014 Jul 18. PMC4194262. ncbi.nlm.nih.gov/pubmed/25046833


MM17-01 Title: Hematopoietic cell transplantation for primary plasma cell leukemia in the era of

novel agents PIs: Saulius Girnius (TriHealth Cancer Institute) Sagar S. Patel (University of Utah) Lohith S. Bachegowda (M.D. Anderson Cancer Center) Binod Dhakal (Medical College of Wisconsin) Status: Submitted (as of July 1, 2019)








Page | 101

MM17-02 Title: The impact of bortezomib based induction therapy vs no induction therapy on

outcomes for light chain amyloidosis PIs: Robert F. Cornell (Vanderbilt University Medical Center) Luciano Costa (University of Alabama at Birmingham) Stacey A. Goodman (Vanderbilt University Medical Center) Status: Protocol Development (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) MM17-03 Title: Assessment of allogeneic hematopoietic cell transplantation in Medicare

beneficiaries with multiple myeloma: A study to develop evidence of effectiveness for the Centers for Medicare and Medicaid Services (CMS)

PIs: Anita D'Souza (Medical College of Wisconsin) Parameswaran Hari (Medical College of Wisconsin) Mary Horowitz (Medical College of Wisconsin) Sergio Giralt (Memorial Sloan Kettering Cancer Center) Gunjun Shah (Memorial Sloan Kettering Cancer Center) Status: Data Collection/Data File Preparation (as of July 1, 2019)

Data Collection/Data File Preparation (expected by June 30, 2020) MM18-01 Title: Racial discrepancy in clinical outcomes of multiple myeloma patients harboring

t(11;14) genetic abnormality PIs: Dharshan Sivaraj (Duke University Medical Center) Amrita Krishnan (City of Hope) Cristina Gasparetto (Duke University Medical Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) MM18-02 Title: Prognostic scoring system of outcomes in patients undergoing autologous stem

cell transplantation for multiple myeloma PIs: Binod Dhakal (Medical College of Wisconsin) Saurabh Chhabra (Medical College of Wisconsin) Natalie Callander (University of Wisconsin Hospital and Clinics) Aric Hall (University of Wisconsin Hospital and Clinics) Zhubin Gahvari (University of Wisconsin) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with EBMT


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MM18-03 Title: To compare the outcomes in young patients with multiple myeloma at diagnosis

(<50 years) undergoing autologous or allogeneic hematopoietic stem cell transplant with older patients (≥50): progression-free and overall survival in a case match

PIs: Pashna Munshi (Georgetown University Hospital) Artur Jurczyszyn (Jagellonion University Medical College) Jan Maciej Zaucha (Medical University of Gdansk) David Vesole (Hackensack University Medical Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) MM18-04 Title: Busulfan, melphalan, and bortezomib versus high-dose melphalan as a

conditioning regimen for autoHCT in MM: Long term follow up of a novel conditioning regimen

PIs: Patrick Hagen (Loyola University Medical Center) Patrick Stiff (Loyola University Medical Center) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) * Collaborative study with Loyola University MM19-01 Title: Impact of induction therapy with VRD vs. VCD on outcomes in patients with

multiple myeloma undergoing stem cell transplantation PIs: Surbhi Sidana (Mayo Clinic) Maxim Norkin (Shands HealthCare & University of Florida) Shaji K. Kumar (Mayo Clinic Rochester) Sergio Giralt (Memorial Sloan Kettering Cancer Center) Status: Draft Protocol Received (as of July 1, 2019)

Analysis (expected by June 30, 2020) MM19-02 Title: Maintenance therapy after second autologous hematopoietic cell transplantation

for multiple myeloma PIs: Oren Pasvolsky (Rabin Medical Center) Moshe Yeshurun (Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital) Uri Rozovski (Rabin Medical Center) Liat Shargian-Alon (Rabin Medical Center) Status: Protocol Pending (as of July 1, 2019)



Page | 103

MM19-03 Title: Second autologous stem cell transplantation as salvage therapy for relapsed or

refractory AL amyloidosis PIs: Carlyn Tan (Fox Chase Cancer Center) Henry Fung (Fox Chase Temple University Hospital Bone Marrow Transplant Program) Status: Draft Protocol Received (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio 16.0 REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE

Page | 104

16.0 REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE

16.1 Leadership

Chair: Shin Mineishi, MD, Penn State Hershey Medical Center Email: [email protected] Chair: Edward Stadtmauer, MD, Abramson Cancer Center University of

Pennsylvania Medical Center Email: [email protected] Chair: Bipin Savani, MD, Vanderbilt University Medical Center Email: [email protected] Scientific Director: Marcelo Pasquini, MD, MS, CIBMTR Milwaukee Email: [email protected] Asst Sci Director: Saurabh Chhabra, MD, CIBMTR Milwaukee Email: [email protected] Statistical Director: Brent Logan, PhD, CIBMTR Milwaukee Email: [email protected] MS Statistician: Mariam Johnson, MPH, CIBMTR Milwaukee Email: [email protected]


2019

Broglie L, Fretham C, Al-Seraihy, George B, Kurtzberg J, Loren A, MacMillan M, Martinez C, Davies SM, Pasquini M. Pulmonary complications in pediatric and adolescent patients following allogeneic hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2019.06.004. Epub 2019 Jun 12. ncbi.nlm.nih.gov/pubmed/31201861

Parikh SH, Satwani P, Ahn KW, Sahr NA, Fretham C, Abraham AA, Agrawal V, Auletta JJ, Abdel-Azim H, Copelan E, Diaz M-A, Dvorak CC, Frangoul HA, Freytes CO, Gadalla SM, Gale RP, George B, Gergis U, Hashmi S, Hematti P, Hildebrandt GC, Keating AK, Lazarus HM, Myers K, Olsson RF, Prestidge T, Rotz S, Savani BN, Shereck E, Williams KM, Wirk B, Pasquini MC, Loren AW. Survival trends in infants undergoing allogeneic hematopoietic cell transplant. JAMA Pediatrics. doi:10.1001/jamapediatrics.2019.0081. Epub 2019 Mar 18. PMC6503511. ncbi.nlm.nih.gov/ pubmed/30882883












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McCune JS, Wang T, Bo-Subait K, Aljurf M, Beitinjaneh A, Bubalo J, Cahn J-Y, Cerny J, Chhabra S, Cumpston A, Dupuis LL, Lazarus HM, Marks DI, Maziarz RT, Norkin M, Prestidge T, Mineishi S, Krem MM, Pasquini M, Martin PJ. Association of antiepileptic medications with outcomes after allogeneic hematopoietic cell transplantation with busulfan / cyclophosphamide conditioning. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2019.03.001. Epub 2019 Mar 11. ncbi.nlm.nih.gov/pubmed/30871976

2018

Thakar MS, Broglie L, Logan B, Artz A, Bunin N, Burroughs LM, Fretham C, Jacobsohn DA, Loren AW, Kurtzberg J, Martinez CA, Mineishi S, Nelson AS, Woolfrey A, Pasquini MC, Sorror ML. The Hematopoietic Cell Transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases. Blood. 2019 Feb 14; 133(7):754-762. doi:10.1182/blood-2018-09-876284. Epub 2018 Dec 13. PMC6376282. ncbi.nlm.nih.gov/ pubmed/30545834

Brunstein CG, Pasquini MC, Kim S, Fei M, Adekola K, Ahmed I, Aljurf M, Agrawal V, Auletta JJ, Battiwalla M, Bejanyan N, Bubalo J, Cerny J, Chee L, Ciurea SO, Freytes C, Gadalla SM, Gale RP, Ganguly S, Hashmi SK, Hematti P, Hildebrandt G, Holmberg LA, Lahoud OB, Landau H, Lazarus HM, de Lima M, Mathews V, Maziarz R, Nishihori T, Norkin M, Olsson R, Reshef R, Rotz S, Savani B, Schouten HC, Seo S, Wirk BM, Yared J, Mineishi S, Rogosheske J, Perales M-A. Effect of conditioning regimen dose reduction in obese patients undergoing autologous hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2019 Mar 1; 25(3):480-487. doi:10.1016/j.bbmt.2018.11.005. Epub 2018 Nov 10. PMC6445718. ncbi.nlm.nih.gov/pubmed/ 30423481

Strouse C, Zhang Y, Zhang M-J, DiGilio A, Pasquini MC, Horowitz MM, Lee S, Ho V, Ramananthan M, Chinratanalab W, Loren A, Burns LJ, Artz A, Villa KF, Saber W. Risk score for development of veno-occlusive disease after allogeneic hematopoietic cell transplant. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2018.06.013. Epub 2018 Jun 18. ncbi.nlm.nih.gov/pubmed/ 29928989

Harris AC, Boelens JJ, Ahn KW, Fei M, Abraham A, Artz A, Dvorak C, Frangoul H, Freytes C, Gale RP, Hong S, Lazarus HM, Loren A, Mineishi S, Nishihori T, O'Brien T, Williams K, Pasquini MC, Levine JE. Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine. Blood Advances. 2018 Jun 12; 2(11):1198-1206. doi:10.1182/bloodadvances.2018016956. Epub 2018 May 29. ncbi.nlm.nih.gov/pubmed/ 29844205

2017

Karaesmen E, Rizvi AA, Preus LM, McCarthy PL, Pasquini MC, Onel K, Zhu X, Spellman S, Haiman CA, Stram DO, Pooler L, Sheng X, Zhu Q, Yan L, Liu Q, Hu Q, Webb A, Brock G, Clay-Gilmour AI, Battaglia S, Tritchler D, Liu S, Hahn T, Sucheston-Campbell LE. Replication and validation of genetic polymorphisms associated with survival after allogeneic blood or marrow transplant. Blood. 2017 Sep 28; 130(13):1585-1596. doi:10.1182/blood-2017-05-784637. Epub 2017 Aug 15. PMC5620418. ncbi.nlm.nih.gov/pubmed/28811306












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Muffly L, Pasquini M, Martens M, Brazauskas R, Zhu X, Adekola K, Aljurf M, Ballen KK, Bajel A, Baron F, Battiwalla M, Beitinjaneh A, Cahn J-Y, Carabasi M, Chen Y-B, Chhabra S, Ciurea S, Copelan E, D'Souza A, Edwards J, Foran J, Freytes CO, Fung HC, Gale RP, Giralt S, Hashmi SK, Hildebrandt GC, Ho V, Jakubowski A, Lazarus H, Luskin MR, Martino R, Maziarz R, McCarthy P, Nishihori T, Olin R, Olsson RF, Pawarode A, Peres E, Rezvani AR, Rizzieri D, Savani BN, Schouten HC, Sabloff M, Seftel M, Seo S, Sorror M, Szer J, Wirk BM, Wood WA, Artz A. Increasing use of allogeneic hematopoietic cell transplantation in patients age 70 years and older in the United States. Blood. 2017 Aug 31; 130(9):1156-1164. doi:10.1182/blood-2017-03-772368. Epub 2017 Jul 3. PMC5580273. ncbi.nlm.nih.gov/pubmed/28674027

D'Souza A, Lee S, Zhu X, Pasquini M. Current use and trends in hematopoietic cell transplantation in the United States. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2017 Sep 1; 23(9):1417-1421. doi:10.1016/j.bbmt.2017.05.035. Epub 2017 Jun 9. PMC5565685. ncbi.nlm.nih.gov/pubmed/ 28606646

2016

Martin PJ, Fan W, Storer BE, Levine DM, Zhao LP, Warren EH, Flowers MED, Lee SJ, Carpenter PA, Boeckh M, Hingorani S, Yan L, Hu Q, Preus L, Liu S, Spellman S, Zhu X, Pasquini M, McCarthy P, Stram D, Sheng X, Pooler L, Haiman CA, Sucheston-Campbell L, Hahn T, Hansen JA. Replication of associations between genetic polymorphisms and chronic graft-versus-host disease. Blood. 2016 Nov 17; 128(20):2450-2456. doi:10.1182/blood-2016-07-728063. Epub 2016 Oct 6. PMC5114491. ncbi.nlm.nih.gov/pubmed/27758874

Artz AS, Logan B, Zhu X, Akpek G, Bufarull RM, Gupta V, Lazarus HM, Litzow M, Loren A, Majhail NS, Maziarz RT, McCarthy P, Popat U, Saber W, Spellman S, Ringden O, Wickrema A, Pasquini MC, Cooke KR. The prognostic value of serum C-reactive protein, ferritin, and albumin prior to allogeneic transplantation for acute myeloid leukemia and myelodysplastic syndromes. Haematologica. 2016 Nov 1; 101(11):1426-1433. doi:10.3324/haematol.2016.145847. Epub 2016 Sep 4. PMC5394859. ncbi.nlm.nih.gov/pubmed/27662010

Magenau J, Westervelt P, Khaled S, McGuirk J, Hari P, Eapen M, Becker PS, Parkin B, Braun T, Logan B, Wang H, Jagasia M, Rowley SD, Kim DD, Schechter T, Frey N, Scott B, Churay T, Lieland S, Forman S, Mineishi S. A multicenter trial of myeloablative clofarabine and busulfan conditioning for relapsed or primary induction failure AML not in remission at the time of allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplantation. 2017 Jan 1; 52(1):59-65. doi:10.1038/bmt.2016.188. Epub 2016 Jul 18. PMC5215562. ncbi.nlm.nih.gov/pubmed/27427921

Pasquini MC, Le-Rademacher J, Zhu X, Artz A, DiPersio J, Fernandez HF, Mineishi S, Kamishohara M, Mehta J, Nakamura Y, Ratanatharathorn V, Sobecks R, Burkart J, Bredeson C. Intravenous busulfan-based myeloablative conditioning regimens prior to hematopoietic cell transplantation for hematologic malignancies. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Aug 1; 22(8):1424-1430. doi: 10.1016/j.bbmt.2016.04.013. Epub 2016 May 3. PMC4949158. ncbi.nlm.nih.gov/pubmed/27154848









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Strouse C, Richardson P, Prentice G, Korman S, Hume R, Nejadnik B, Horowitz MM, Saber W. Defibrotide for treatment of severe veno-occlusive disease in pediatrics and adults: An exploratory analysis using data from the CIBMTR. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2016 Jul 1; 22(7):1306-1312. doi:10.1016/j.bbmt.2016.04.011. Epub 2016 Apr 19. PMC4914048. ncbi.nlm.nih.gov/pubmed/27108694

2015

Hahn T, Sucheston-Campbell LE, Preus L, Zhu X, Hansen JA, Martin PJ, Yan L, Liu S, Spellman S, Tritchler D, Clay A, Onel K, Pasquini M, McCarthy PL. Establishment of definitions and review process for consistent adjudication of cause-specific mortality after allogeneic unrelated-donor hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):1679-1686. doi:10.1016/j.bbmt.2015.05.019. Epub 2015 May 29. PMC4537799. ncbi.nlm.nih.gov/ pubmed/26028504

Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OTH, Pasquini MC, Schriber JR, Cooke KR. Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies. Leukemia. 2015 Aug 1; 29(8):1754-1762. doi:10.1038/leu.2015.75. Epub 2015 Apr 24. PMC4527886. ncbi.nlm.nih.gov/pubmed/25772027

Sorror ML, Logan BR, Zhu X, Rizzo JD, Cooke KR, McCarthy PL, Ho VT, Horowitz MM, Pasquini MC. Prospective validation of the predictive power of the hematopoietic cell transplantation comorbidity index: A Center for International Blood and Marrow Transplant Research study. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):1479-1487. doi:10.1016/j.bbmt.2015.04.004. Epub 2015 Apr 7. PMC4512746. ncbi.nlm.nih.gov/pubmed/25862591

Holter-Chakrabarty JL, Pierson N, Zhang M-J, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL. The sequence of cyclophosphamide and myeloablative total body irradiation in hematopoietic cell transplantation for patients with acute leukemia. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1251-1257. doi:10.1016/j.bbmt.2015.03.017. Epub 2015 Mar 31. PMC4465990. ncbi.nlm.nih.gov/pubmed/25840335

Chen Y-B, Lane AA, Logan BR, Zhu X, Akpek G, Aljurf MD, Artz AS, Bredeson CN, Cooke KR, Ho VT, Lazarus HM, Olsson RF, Saber W, McCarthy PL, Pasquini MC. Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous hematopoietic cell transplantation. Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. doi:10.1016/j.bbmt.2015.02.005. Epub 2015 Feb 13. PMC4426014. ncbi.nlm.nih.gov/pubmed/ 25687795










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2014

Hong S, Le-Rademacher J, Artz A, McCarthy PL, Logan BR, Pasquini MC. Comparison of non-myeloablative conditioning regimens for lymphoproliferative disorders. Bone Marrow Transplantation. 2015 Mar 1; 50(3):367-374. doi:10.1038/bmt.2014.269. Epub 2014 Dec 1. PMCID4351124. ncbi.nlm.nih.gov/pubmed/25437248


RT13-02 Title: Safety of high-dose TBI followed by an allogeneic stem cell transplant for

hematologic malignancies PI: Mitchell Sabloff (The Ottawa Hospital Blood & Marrow Transplant Program) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) RT14-02 Title: Endothelial injury complications after allogeneic hemotapoietic cell

transplantation PIs: Narendranath Epperla (Ohio State Medical Center, James Cancer Center) Ang Li (University of Washington) Status: Manuscript Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) RT14-03 Title: Multicenter cohort identification of transplant-related risk-factors for infection,

organ failure, and mortality among pediatric hematopoietic stem cell transplant patients requiring intensive care unit admission

PIs: Matt Zinter (University of California San Francisco Medical Center) Chris Dvorak (University of California San Francisco Medical Center) Anil Sapru (University of California San Francisco Medical Center) Status: Submitted (as of July 1, 2019)

Published (expected by June 30, 2020) * Collaborative study with Virtual Pediatric Systems RT17-01 Title: Allogeneic hematopoietic stem cell transplant outcome for patients with end

stage renal disease on dialysis PIs: Nosha Farhadfar (Shands HealthCare & University of Florida) Ajoy Dias (University of Kansas Medical Center) John Reid Wingard (Shands HealthCare & University of Florida) Hemant Murthy (Mayo Clinic Florida) Sid Ganguly (University of Kansas Medical Center) Status: Manuscript Preparation (as of July 1, 2019)




Page | 109

RT18-01 Title: Developing a modified hematopoietic stem cell transplantation-comorbidity

index for adolescents and young adults / A modified hematopoietic cell transplantation-comorbidity index (HCT-CI) for pediatric recipients of allogeneic transplantation

PIs: Brian Friend (University of California Los Angeles) Gary Schiller (UCLA Health) Larisa Broglie (Columbia University) Monica Thakar (Fred Hutchinson Cancer Research Center) Mohamed Sorror (Fred Hutchinson Cancer Research Center) Status: Data File Preparation (as of July 1, 2019)

Submitted (expected by June 30, 2020) RT18-02 Title: The effect of obesity on outcomes after alternative donor stem cell transplants PIs: Mouhamed Yazan-Abou Ismail (Rochester General Hospital) Gayathri Ravi (Seidman Cancer Center - University Hospitals Cleveland Medical Center) Leland Metheny (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) RT18-03 Title: An analysis of non-infectious pulmonary toxicities in myeloablative total body

irradiation vs chemotherapy-based conditioning regimens after alloHCT for hematologic malignancies / Diffuse alveolar hemorrhage (DAH) is a result of complex interaction between conditioning regimen (MAC>RIC and TBI>noTBI), graft source (UCB >others), and engraftment (delayed>early).

PIs: Sagar Patel (Cleveland Clinic Foundation) Betty Hamilton (Cleveland Clinic Foundation) Navneet Majhail (Cleveland Clinic Foundation) Celattin Ustun (The Coleman Foundation Blood and Marrow Transplant Center, Rush

University) Status: Data File Preparation (as of July 1, 2019)

Manuscript Preparation (expected by June 30, 2020) RT19-01 Title: Analysis of comorbidity-associated toxicity at the regimen level. PIs: Roni Shouval (Sheba Medical Center) Bipin Savani (Vanderbilt University Medical Center) Aarnon Nagler (Sheba Medical Center) Status: Draft Protocol Received (as of July 1, 2019)



Page | 110

RT19-02 Title: Hemorrhagic cystitis as a complication of HCT in the Pt-Cy GVHD prophylaxis era

compared to other alloHCTs. PIs: Kehinde Adekola (Northwestern Medicine) Naveed Ali (Federal Institute for Material Research) Olga Frankfurt (Northwestern Medicine) Leland Metheny (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Jonathan Moreira (Northwestern Medicine) Marcos de Lima (Seidman Cancer Center-University Hospitals Cleveland Medical

Center) Status: Protocol Pending (as of July 1, 2019)


2019 CIBMTR Scientific Working Committee Research Portfolio APPENDIX A: COLLABORATIVE STUDIES

Page | 111

APPENDIX A: COLLABORATIVE STUDIES

AABB

• DS17-02: Impact of pre-operative collection of auto blood for BM harvest on donor health and outcome

o Also in collaboration with ASTCT

ASTCT

• DS17-02: Impact of pre-operative collection of auto blood for BM harvest on donor health and outcome

o Also in collaboration with AABB

Brazilian Transplant Group

• HS19-03: Haploidentical stem cell transplantation for malignant and non-malignant hematological diseases in patients without sibling donor: A multicenter prospective longitudinal study of the Brazilian bone marrow transplantation study group

• HS19-04: Outcomes after allogeneic stem cell transplants performed in Brazil from HLA-matched siblings, unrelated and mismatched related donors: Retrospective study on behalf of the Brazilian Bone Marrow Transplantation Society (SBTMO), GEDECo (Brazil-Seattle Transplant-related complications Consortium), Hospital Israelita Albert Einstein (AmigoH), Associação da Medula Óssea do Estado de São Paulo (Ameo), Program Nacional de Apoio à Atenção Oncológica (Pronon), and CIBMTR

CALGB 10403

• LK15-03: Comparison of outcomes of older adolescents and young adults with Philadelphia-chromosome / BCR-ABL1-negative acute lymphoblastic leukemia receiving post-remission consolidation chemotherapy with pediatric-inspired chemotherapy on CALGB 10403 or myeloablative allogeneic hematopoietic cell transplantation

City of Hope

• CK15-01: Comparison of transplant vs non transplant therapies for myelofibrosis o Also in collaboration with 12 other organizations

Cleveland Clinic Foundation


Dana-Farber Cancer Institute



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Deutsche Knochenmarkspenderdatei GmbH (DKMS, German Bone Marrow Donor Center)

• IB18-04: Evaluation of the impact of donor KIR genotype on outcome after unrelated donor transplantation in patients with myelodysplastic syndromes or secondary acute myeloid leukemia

Duke University Medical Center


EBMT

• CK17-01: Development of a prognostic scoring system predictive of outcomes in patients with myelofibrosis after allogeneic hematopoietic cell transplantation

• CK19-01b: Outcomes after HCT for rare chronic leukemias: Outcomes of chronic neutrophilic leukemia patients who underwent alloHCT

• GS18-03: Comparison of outcomes of reduced intensity transplantation in lymphoma patients using haploidentical related donors vs. unrelated cord blood

• LY19-01: Post-transplant cyclophosphamide-based haploidentical transplantation versus matched sibling or well-matched unrelated donor transplantation for peripheral T-cell lymphoma: A CIBMTR Lymphoma Working Committee and EBMT Lymphoma Working Party analysis

• NM15-01: Outcome of allogeneic hematopoietic cell transplant in erythropoietic porphyria • NM17-01: Late effects after hematopoietic stem cell transplantation in patients with

hemophagocytic lymphohistiocytosis • PC19-03: The impact of pre-transplant extramedullary disease on the outcome of

allogeneic hematopoietic cell transplantation for acute myeloid leukemia in children: A combined CIBMTR and EBMT analysis

• MM18-02: Prognostic scoring system of outcomes in patients undergoing autologous stem cell transplantation for multiple myeloma

The Emmes Company

• LE18-03: Incorporating patient reported outcomes into individualized prognostication tools for survival and quality of life in transplant patients

Emory University

• LE17-01: Long-term follow up after hematopoietic stem cell transplantation for sickle cell disease

H. Lee Moffitt Cancer Center and Research Institute


The Hospital for Sick Children

• IB19-05: Impact of donor signal-regulatory protein alpha polymorphism on outcomes of allogeneic hematopoietic stem cell transplantation


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International Histocompatibility Working Group

• IB06-05: Use of high-resolution HLA data from the NMDP for the International Histocompatibility Working Group in HCT

• IB09-01: Clinical importance of minor histocompatibility complex haplotypes in umbilical cord blood transplantation

• IB09-03: Clinical relevance of cytokine / immune response gene polymorphisms in umbilical cord blood transplantation

• IB09-05: Identification of functional single nucleotide polymorphisms in umbilical cord blood transplantation

• IB09-07: Clinical significance of genome-wide variation in unrelated HCT • IB14-07: Indirectly recognizable HLA epitopes (PIRCHES): A retrospective validation study on

the role of indirect recognition of mismatched HLA in hematopoietic stem cell transplantation outcome

• IB18-05: Imputation of KIR in genome-wide association study and the association of KIR-HLA with outcomes following alloHCT In AML and MDS

• R04-74d: Functional significance of killer cell immunoglobulin-like receptor genes in HLA-matched and mismatched unrelated HCT

Japan Society for Hematopoietic Cell Transplantation

• HS18-01: International collaborative study to compare the prognosis for acute leukemia patients transplanted with intensified myeloablative regimens

Loyola University

• MM18-04: Busulfan, melphalan, and bortezomib versus high-dose melphalan as a conditioning regimen for AutoHCT in MM: Long term follow up of a novel conditioning regimen

M.D. Anderson Cancer Center

• CK12-01: Optimal timing of allogeneic stem cell transplantation for chronic myeloid leukemia patients in the tyrosine kinase inhibitor era


Massachusetts General Hospital


Mayo Clinic Arizona and Phoenix Children's Hospital



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Medical College of Wisconsin


• IB15-04: Clinical outcomes among hematopoietic stem cell transplant recipients as a function of socioeconomic status and related transcriptome differences

Memorial Sloan Kettering Cancer Center


National Cancer Institute

• IB10-01f: Epigenetic clock: Can this guide donor selection in HCT? • IB16-03: Role of recipient and donor genetic polymorphisms in interferon lambda 4

(INFL4) on outcomes after unrelated allogeneic cell transplant • IB17-03: Identification of genomic markers of post hematopoietic cell transplantation

(HCT) outcomes in patients with myelofibrosis: A pilot study • IB18-06: Clonal mosaicism and HCT outcomes in patients with acute leukemia and

myelodysplastic syndromes • IB18-07: Donor and recipient genomic associations with acute GVHD • AA13-02: Malignancies in patients with Fanconi anemia

National Heart, Lung, and Blood Institute

• NM19-01: Conditional and cause-specific mortality of patients with severe aplastic anemia surviving at least one year after alloHCT or immunosuppressive therapy

National Institutes of Health

• LE13-02: Risk factors for melanoma following allogeneic hematopoietic stem cell transplantation

Northwestern University


The Ohio State University

• IB09-06f: DISCOVeRY-BMT: Case-control study of acute myelogenous leukemia exome and genome-wide association study

• IB09-06j: DISCOVeRY-BMT: Additional analysis of major histocompatibility complex single nucleotide polymorphisms

• IB09-06m: DISCOVeRY-BMT: Analysis of X chromosome single nucleotide polymorphisms • IB09-06n: DISCOVeRY-BMT: Compare unrelated donor to Welcome Trust Case Control

Consortium controls • IB09-06o: DISCOVeRY-BMT: Genetics and epidemiology of myeloid malignancies

candidate gene paper


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• IB09-06p: DISCOVeRY-BMT: Genetics and epidemiology of myeloid malignancies genome-wide association study

• RT09-04 / IB09-06i: DISCOVeRY-BMT: Recip, donor genome-wide association study interaction with conditioning intensity (myeloablative / reduced intensity conditioning), total body irradiation, disease status

Peking University

• HS19-02: Comparing outcomes of myeloablative T-replete haploidentical transplantation with PT-CY protocol and ATG+G-CSF protocol in patients with cytogenetic intermediate / high risk acute myeloid leukemia in first complete remission

Pediatric Health Information System (PHIS)

• HS14-01: Investigating clinical outcomes and inpatient health care resource utilization of hematopoietic cell transplantation for children with acute leukemia

• HS17-01: Association of community health status and center survival for allogeneic hematopoietic cell transplantation

• HS18-03: Racial / ethnic disparities in receipt of hematopoietic cell transplantation and subsequent resource utilization in children with acute leukemia

Roswell Park Cancer Institute

• IB09-06b: Genetic susceptibility to transplant-related mortality after unrelated donor stem cell transplant

• IB09-06f: DISCOVeRY-BMT: Case-control study of acute myelogenous leukemia exome and genome-wide association study

• IB09-06j: DISCOVeRY-BMT: Additional analysis of major histocompatibility complex single nucleotide polymorphisms

• IB09-06m: DISCOVeRY-BMT: Analysis of X chromosome single nucleotide polymorphisms • IB09-06n: DISCOVeRY-BMT: Compare unrelated donor to Welcome Trust Case Control

Consortium controls • IB09-06o: DISCOVeRY-BMT: Genetics and epidemiology of myeloid malignancies

candidate gene paper • IB09-06p: DISCOVeRY-BMT: Genetics and epidemiology of myeloid malignancies genome-

wide association study • RT09-04 / IB09-06i: DISCOVeRY-BMT: Recip, donor genome-wide association study

interaction with conditioning intensity (myeloablative / reduced intensity conditioning), total body irradiation, disease status

Severe Chronic Neutropenia International Registry

• ID13-01: Haematopoietic stem cell transplants for congenital neutropenia / Kostmann agranulocytosis

University of Florida

• LE99-01: Quality of life in late HCT survivors


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University of Chicago

• CK16-01: Identification of germline predisposition mutations in young myelodysplastic syndrome patients

University of Michigan


University of Minnesota

• IB14-05: mtDNA haplotypes and unrelated donor transplant outcomes • IB15-03: Killer immunoglobulin receptor (KIR) gene content and pediatric acute leukemia

transplant outcomes • R02-40 / R03-63d: Acquisition of natural killer cell receptors in recipients of unrelated

transplant • SC09-05d: Regret having bone marrow transplant

University of Pittsburgh

• DS19-02: The impact of pre-apheresis health related quality of life on peripheral blood progenitor cells yield and donor’s health and outcome

University of Utah

• DS05-02d: QoL for related adult donors compared to unrelated adult donors • DS05-02g: Late toxicities and SAE for related donors

United Network for Organ Sharing

• LE12-03: Solid organ transplantation and hematopoietic cell transplantation

Virtual Pediatric Systems

• RT14-03: Multicenter cohort identification of transplant-related risk-factors for infection, organ failure, and mortality among pediatric hematopoietic stem cell transplant patients requiring intensive care unit admission

• LE19-01: Long-term survival and late effects in critically ill pediatric hematopoietic cell transplant patients

Washington University


• DS18-01: To quantify age-related clonal hematopoiesis in healthy marrow and blood donors that may affect the clinical outcome of recipients following HCT

2019 CIBMTR Scientific Working Committee Research Portfolio APPENDIX B: STUDY DEVELOPMENT AND MANAGEMENT PROCESS

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APPENDIX B: STUDY DEVELOPMENT AND MANAGEMENT PROCESS This study development cycle pertains to studies for which CIBMTR provides data, scientific, and statistical support. Data sets are also made available to investigators who have their own statistical resources. Manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission.

STUDY DEVELOPMENT AND MANAGEMENT PROCESS

Planned Protocol pending. Proposals remain in this preliminary stage until the principal investigator (PI) creates a draft protocol.

In Progress

Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it.

Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee.

Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome.

Supplemental forms / data collection. Most studies use routinely-collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on centers and supplement forms reimbursement costs.


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In Progress (continued)

Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow-up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS-level statistician, working with the Scientific Director, PI(s), and sometimes the Clinical Research Coordinator, to ensure data quality: • Verifying selection criteria • Including and excluding patients so that the investigators can determine

whether the final study population is representative of the target population

• Assessing follow-up • Determining the extent and nature of missing values and their potential

effect on the study • Resolving and reconciling data discrepancies / outliers by examining data

collection forms and communicating with centers and the PI

Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Study PI(s) and associated Working Committee Chairs present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript.

Ongoing. A study in ongoing status is long-term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study typically has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year.

Preliminary Results

Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. Study Leadership reviews and revises the document, ensuring that the description and interpretation of the statistical analyses are accurate and contribute to the fundamental message of the manuscript. The Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript.


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Preliminary Results

(continued)

Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician forwards all editor and reviewer comments to the PI and Statistical Director. The PI is expected to prepare a response, working with Study Leadership who provide additional analyses of data, as needed. Coordinating Center personnel communicate with the journal, including re-submissions, in most cases.

In press. A publication is in press when it has been approved but does not yet have a citation.

Completed Published. A manuscript is considered published when a citation is available, including a PMCID number, if applicable.

2019 CIBMTR Scientific Working Committee Research Portfolio APPENDIX C: GUIDELINES FOR STUDY PIS

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APPENDIX C: GUIDELINES FOR STUDY PRINCIPAL INVESTIGATORS The role of a Study Chair / PI is simple: always behave ethically and do whatever it takes to complete the study that answers your research question. It is easier to accomplish this task if you have an understanding of the CIBMTR study process, specifically where and when your efforts are most needed. The following document will explain the life-cycle of a CIBMTR observational study and review the responsibilities of a PI. Hints and tips to make the study process as successful as possible are noted with an arrow (→).

STUDY PROPOSAL

The PI is usually the first person who suggests the study and who prepares the Study Proposal (cibmtr.org/Studies/Observational/ProposeStudy/Pages/index.aspx). Ideally the PI presents his or her proposal in person to the appropriate Working Committee at the TCT Meetings. Some PIs view the CIBMTR presentation as a formality but, in reality, it is an important opportunity to convince the other Working Committee members that your study is more important, more feasible, and more likely to advance the field and be published in a high-profile journal than other studies being proposed. CIBMTR Working Committee hours and resources are limited, and not all good studies proposed can be supported.

→ Review the data collection forms (cibmtr.org/DataManagement/DataCollectionForms/Pages/ Investigators.aspx) to ensure the data you wish to study are available for the timeframe you wish to study. Many people propose studies that require data not collected routinely by the CIBMTR. Studies that require additional data collection usually get greater scrutiny because of the extra time and effort required whenever centers have to be contacted for additional data. Additionally, the response rate to requests for supplemental data is often disappointing; ask your own data managers how difficult it is to go back and find data for patients transplanted years ago. The ability to collect supplemental data successfully depends on how complex and / or extensive the data are, the size of the study population, how far back in time the transplants were done, and whether you have resources (people or funds) to assist in the process.

→ Examine the latest accrual tables for your proposed Working Committee to ensure there are sufficient data in the CIBMTR Research Database to answer your study questions. Accrual tables are provided in the meeting agenda for each committee’s annual in person meeting at the TCT Meetings under Attachment 2.

→ Review this report and / or the CIBMTR Publication List and Working Committee Study Lists of planned, in progress, and recently published studies to ensure someone else has not already conducted the study you are proposing.

→ Note that study proposals may be submitted throughout the year. The vast majority are submitted just before the deadline, three months before the TCT Meetings. If you want your proposal to benefit from greater CIBMTR statistical and scientific input, then submitting your proposal far in advance is helpful. Proposals submitted throughout the year will be reviewed by the Working Committee Chairs and Scientific Director. They have the authority to approve a proposal based on the importance of the scientific question or they may elect to defer it until presentation at the Working Committee meeting.

https://www.cibmtr.org/Studies/Observational/ProposeStudy/Pages/index.aspx

https://www.cibmtr.org/DataManagement/DataCollectionForms/Pages/Investigators.aspx

https://www.cibmtr.org/DataManagement/DataCollectionForms/Pages/Investigators.aspx

https://www.cibmtr.org/Meetings/Materials/WorkingCommittees/pages/index.aspx


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PRIORITIZATION AND DISTRIBUTION OF STATISTICAL HOURS

After the TCT Meetings, the Working Committee Chairs, Scientific Directors, Statistical Director, and MS Statistician meet to discuss the results of the meeting and prioritize new and ongoing studies. Studies are assigned Coordinating Center hours according to their need and priority. In general, a study needs 100 hours of Statistical Staff time to finish the protocol document, 100-160 hours to prepare the data file (depending on whether additional data collection, follow-up, or excessive data cleaning is necessary), 60 hours for the analysis phase, and 70 hours for manuscript preparation. PIs are generally notified about Committee decisions (i.e., approval, prioritization, and assigned hours) regarding their proposals within one month after the meeting. At that time, PIs also learn which MS-level Statistician is assigned to their studies; this MS-level Statistician subsequently serves as the point person for communications regarding study issues.

→ PIs can increase the chance of their proposal being approved by carefully preparing the Proposal Form that is presented to the Working Committee. Discussion with Working Committee Leadership in advance of the Working Committee meeting may help clarify the study and address study design questions. Many great concepts fail because PIs do not consider available data, size of the available study population, power calculations, and other statistical issues. The Working Committee is much less receptive to studies that appear to have multiple unresolved issues at the meeting.

STUDY PROTOCOL

The next step in the study’s life is generation of the study protocol. This is an important document that is first drafted by the PI and submitted to the MS-level Statistician. The draft study protocol should be completed within two months of concept approval notification. In preparing this document, it is crucial to carefully review the applied study selection criteria and description of patient, disease, and transplant characteristics. The PI must also carefully consider the variables to be included in the analysis because the MS-level Statisticians, Statistical Directors, and Scientific Directors use these documents to guide data collection and cleaning. Common pitfalls include failure to include important variables to address study hypotheses and failure to consider potentially confounding variables. After the initial draft is reviewed and approved by the Coordinating Center, it is circulated to the Working Committee for comment; at that time, Committee members may request to participate in the study and a Writing Committee is formed (see below). Individuals wishing to serve on the Writing Committee provide substantive comments on the study protocol. It is the PI’s responsibility to collate and address these comments by either modifying the protocol or providing an explanation for not incorporating suggested changes. Since Writing Committee members earn their authorship by reviewing the study protocol, analyses, and manuscripts, the CIBMTR also keeps track of comments and contributions.

→ Each study protocol is reviewed at the weekly Coordinating Center conference call / meeting (held on Tuesdays, 9:00-10:00 am US Central Time) before distribution to the Working Committee; it is very helpful for the PI to join that meeting by phone and to participate in the discussion of the study’s design and implementation. (Studies are again discussed at a Coordinating Center weekly meeting as they reach significant milestones. PI participation in each of these discussions is strongly encouraged.)


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→ The most successful PIs respond to Writing Committee critiques as they do journal reviews — by carefully organizing them and responding to each. If a Writing Committee member brought up an issue, it is likely that a reviewer will also bring up the same points. It is expected that the PI will summarize and respond to these critiques within three weeks after the deadline for comments has passed.

→ PIs have a great deal of control over the interval between study proposal approval and the completion of a final study protocol. Timely submission of the draft protocol and response to Writing Committee comments can vault your study ahead of others in terms of Coordinating Center priority. If yours is ready to go and another is not, yours may be given priority, even if initially it was planned for the other study to be done first.

DATA COLLECTION

If supplemental data collection is needed for the study, approval from the Chief Scientific Director is required. The PI needs to provide the following information for the approval: 1) number of questions, 2) types of questions, 3) number of cases and 4) the study calendar. Once the request has been approved, the Forms Development Clinical Research Coordinator will prepare a supplemental form for review within one week. This draft form will be a Word document listing all the supplemental questions that are relevant, as well as the most frequent response options. This form will have input from the Scientific Director, PI, Study Statistician, Metadata and Data Operations Staff for clarity, length, internal consistency of response options, and feasibility of data retrieval. The form will be formatted to be consistent with other CIBMTR forms, and a table will be created in the database to receive the data. This step is very important for any study collecting additional data. If the form is long or leaves out critical variables, the ultimate study results could be compromised by missing data. The supplemental form will go to the Chief Scientific Director, Scientific Director, and PI for final approval. The Scientific Director and PI will prepare a letter detailing the importance of the data needed for the study with a copy to the Medical Director. This letter will be sent with the study request. If terms or concepts on the supplemental data collection form are unfamiliar to the data management teams, an instruction manual that describes the variable and provides examples of how data managers should interpret primary data will have to be written. Each study is assigned a Clinical Research Coordinator who communicates with centers to facilitate data submission. Most, but not all, centers are very responsive to these requests. If some centers are lagging behind in submitting extra forms, PIs may need to make personal email or phone appeals.

→ Providing the initial draft form and content for the instruction manual is the responsibility of the PI. Delay in putting it together can significantly delay initiating the data collection process. If the process is inordinately delayed so that the data needed for a study is not available in a timely manner, the study may be deferred to the next year.

→ For smaller studies, where every patient counts, personal appeals from the PI to the Transplant Center Director can sometimes be very effective.

DATA FILE PREPARATION

In this step, the MS-level Statistician prepares a data file using the finalized study protocol as guidance. Data interpretation issues may arise here, especially if uncommon variables are necessary for the study. Values for common variables have probably already been reviewed and, if missing or out of range or inconsistent, already clarified (data “cleaning”) for other studies. If


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your study is the first to examine a particular variable or study population, then expect to do a lot of data cleaning.

→ The PI can accelerate this process by being available to the MS-level Statistician and Scientific Director as questions come up. The PI should also carefully review the frequencies of study variables for outliers and other clinical inconsistencies.

UNIVARIATE ANALYSIS

Once the data file is prepared, the MS-level Statistician performs as much of the analysis as possible before handing the data set to the Statistical Director assigned to the project. First, a table of study population characteristics and preliminary univariate analysis is prepared. This is reviewed by the PI and Scientific Director. When they are satisfied with the population, the study is scheduled for another Coordinating Center weekly meeting / conference call to confirm final composition of the population and study design and review the univariate analysis before multivariate analyses are performed. Relevant comments from the Coordinating Center review will be summarized by the MS-level Statistician or the Scientific Director and relayed back to the PI for comment if the PI cannot participate personally in the meeting.

→ As noted above, the PI is invited to participate in the CIBMTR Coordinating Center Meeting (Tuesdays) when his or her study is discussed. It is worth repeating that it is very helpful for the PI to participate since they can often address questions as they arise so that the statistical input is most helpful.

MULTIVARIATE ANALYSIS

Once the population characteristics and univariate analyses are approved, the data file is transmitted to the Statistical Directors for multivariate and more complex modeling. When completed, results are sent to the PI and Scientific Director who present them on a weekly Coordinating Center conference call. The PI and Scientific Director address comments provided at the meeting and then prepare a memo for circulation to the Writing Committee for comments. The comment period usually lasts two to three weeks. The PI summarizes the comments and prepares another memo for the Writing Committee within three weeks of the close of the comment period. If substantive issues arise, especially related to the study population or analyses, then a conference call involving the PI, Scientific Director, Statistical Director, and MS-level Statistician may need to be convened to plan an approach for addressing the comments.

→ The most successful PIs take advantage of the MS-level Statisticians’ and Statistical Directors’ familiarity with the project and the data to finish their analyses quickly. If extended time passes between each phase of the analysis, the Statisticians will have to re-familiarize themselves with the project and coding. A task that could take a couple of hours immediately after the initial results are completed may take much longer a month or two later (and the Statisticians understandably will be less excited about picking up the project again).

ABSTRACTS

Many PIs hope to submit abstracts to national and international meetings. Multivariate analyses must be complete with enough time to allow generation of an abstract. These abstracts must be circulated to the Writing Committee and reviewed by the Coordinating Center faculty and staff prior to submission. Please allow enough time to complete these steps before the abstract


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deadline. If the abstract is accepted for oral presentation, the Coordinating Center staff will also need to review the slides, primarily for accuracy but sometimes also to make suggestions for clarity. The CIBMTR has a template for format and background that is required for all presentations.

→ Planning for meeting abstracts for the American Society of Hematology and other meetings happens immediately after the TCT Meetings. If you would like to submit your abstract to one of these meetings, an early declaration of your intentions and demonstrable effort in moving towards that goal will result in your study getting higher priority.

→ In general, studies are only submitted to one meeting; once submitted in abstract form, priority should be placed on writing and submitting the manuscript.

MANUSCRIPT

Once the analysis is completed, drafting the manuscript is the responsibility of the PI. A draft manuscript is expected within 30 days of the final analysis. The draft is circulated to the Writing Committee and comments are again summarized and incorporated. At least one round and sometimes up to three or more rounds are necessary to create a final manuscript. The CIBMTR will do the final formatting for journal submission, attach all the co-authors’ information (such as institution and contact information), collect any necessary signatures, and submit the paper. The CIBMTR has a long list of acknowledgment for funding sources that are attached to the paper.

→ The initial manuscript draft usually causes the greatest delay in study progress and is the step most directly under control of the PI. The most successful PIs recognize that publishing their study results is a critical measure of success for all involved parties - themselves, the CIBMTR and all the collaborators involved in the study. Working Committee Chairs have the authority to re-assign a study to a different PI if the delay in manuscript preparation is too long (>60 days).

ACCEPTANCE

Unless the paper is accepted on the first submission, it will need to be revised or resubmitted. If comments are straightforward, the PI can prepare a response to reviewers for circulation, along with the revised version. Some comments from reviewers require additional analyses or discussion at a Coordinating Center meeting prior to resubmission. The CIBMTR will assist with manuscript resubmission. Once the paper is accepted, the PI also handles proof review.

→ Unless a study is completed in record time, it will be “in progress” at the next TCT Meetings. PIs should plan to present a study update at the CIBMTR Working Committee meetings or designate another person on the Writing Committee to do this, as long as the study is active.

→ Any expected or unexpected deviations from the above timetable should be discussed between the PI and Working Committee Leadership. Sometimes unavoidable delays are due to either the CIBMTR or the PI. A proactive plan designed to keep the study moving forward should be devised. Generally, the CIBMTR expects studies to be completed within 18-24 months.

2019 CIBMTR Scientific Working Committee Research Portfolio APPENDIX D: GLOSSARY

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APPENDIX D: GLOSSARY Abbreviation/

Acronym Meaning

AA aplastic anemia AABB American Association of Blood Banks ABO Landsteiner's blood grouping system ALL acute lymphoblastic leukemia alloHCT allogeneic hematopoietic cell transplantation AML acute myeloid (myelogenous) leukemia APL acute promyelocytic leukemia ASTCT American Society for Transplantation and Cellular Therapy (formerly

ASBMT – American Society for Blood and Marrow Transplantation) autoHCT autologous hematopoietic cell transplantation BM bone marrow BMT bone or blood marrow transplant BMT CTN Blood and Marrow Transplant Clinical Trials Network BSI bacterial bloodstream infections CALGB Cancer and Leukemia Group B (member Alliance for Clinical Trials in

Oncology) CIBMTR Center for International Blood and Marrow Transplant Research CMV cytomegalovirus CR2 second complete remission CRF Comprehensive Report Form DLBCL diffuse large B-cell lymphoma DNA deoxyribonucleic acid DRI disease risk index EBMT European Society for Blood and Marrow Transplantation ECOG Eastern Cooperative Oncology Group FA Fanconi anemia FACT Foundation for the Accreditation of Cellular Therapy FL follicular lymphoma FLT3 FMS like tyrosine kinase 3 G-CSF subcutaneous filgrastim GVHD graft-versus-host disease HCT or HSCT hematopoietic stem cell transplantation HLH hemophagocytic lymphohistiocytosis JACIE Joint Accreditation Committee – International Society for Cellular Therapy &

European Society for Blood and Marrow Transplantation JMML Juvenile Myelomonocytic Leukemia KIR killer-cell immunoglobulin-like receptors KIR3DL1 killer cell immunoglobulin like receptor, three Ig domains and long

cytoplasmic tail 1 MDS myelodysplastic syndrome mtDNA mitochondrial DNA

2019 CIBMTR Scientific Working Committee Research Portfolio APPENDIX D: GLOSSARY

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Abbreviation/ Acronym Meaning

MHC major histocompatibility complex MLL mixed lineage leukemia MS Master of Science (level statistician) NK natural killer (cell) NIH National Institutes of Health NIMA National Integrated Medical Association NMDP National Marrow Donor Program PBSC peripheral blood stem cell PBSCT peripheral blood stem cell transplantation PHIS Pediatric Health Information System® PI principal investigator PICU pediatric intensive care unit PIRCHES predicted indirectly recognizable HLA epitopes PMCID PubMed Central unique identifier QoL quality of life SAE severe adverse event SWOG Southwest Oncology Group SWOT strengths, weaknesses, opportunities, threats TBI total body irradiation TNFSF4 tumor necrosis factor superfamily member 4 TRM transplantation-related mortality US United States VRE vancomycin-resistant enterococcus vs versus

Documents

CIBMTR Scientific July 1, Working Committee 2019 ...• If Working Committee Leadership clears the proposal to move forward, the MS-level Statistician contacts the study investigator