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PEDIATRIC RESEARCH NEWSLETTERPEDIATRIC RESEARCH NEWSLETTERPEDIATRIC RESEARCH NEWSLETTER Volume 01 July 2012

Denny Hamlin Foundation Pledges Support for CF Research

Denny Hamlin, FedEx #11 NASCAR Sprint Cup Driver, announced today that the Denny Hamlin Foundation will give $150,000 to Chil-dren’s Hospital of Richmond at VCU and the Cystic Fibrosis Foundation to help fund cystic fibrosis research and clinical trials for cystic fibrosis in Richmond.

“This is a big day for the Denny Hamlin Foun-dation,” Hamlin said. “It is exactly what I envi-sioned the foundation doing when I started it five years ago, and we’ve worked hard to get to a point where we can give this kind of gift.

“VCU is one of the country’s leading research centers for CF, and it is right here in our own backyard,” Hamlin said. “I am honored to sup-port such an outstanding organization with people who are making a difference in the world.”

The Denny Hamlin Foundation will give $50,000 for the next three years for CF re-search and to support the Children’s Hospital of Richmond at VCU Therapeutics Develop-ment Center, which conducts all phases of clinical trials for potential CF therapies.

“I have been caring for children and adults with cystic fibrosis for more than 35 years and my laboratory has been studying CF and de-veloping new therapies for over 25 years,” said Bruce Rubin, M.D., professor and chair of

the VCU Department of Pediatrics and physi-cian-in-chief of Children’s Hospital of Rich-mond at VCU. “This generous donation will help accelerate our ability to bring these thera-pies to our patients, and the newly established Denny Hamlin Foundation Summer Scholars program will help us train the next generation of CF research scientists.”

CF is a genetic disease that affects about 30,000 children and adults in the United States and about 70,000 worldwide. CF causes chronic infections in the lungs and inadequate digestion of nutrients. There is no cure for CF, but medical advances have dou-bled the life expectancy of children in the last 30 years and increased the overall median age of survival to 38. According to Rubin, CF research and care has much in common with NASCAR racing. “It is the talented pit crew that makes sure it all works. My pit crew is the many amazing scien-tists in the labs and the fantastic clinical care team. And just as sponsors keep the wheels on the car and gas in the tank, sponsorship of our CF care and research center by the Denny Hamlin Foundation will allow us to compete at the international level as we race to find a cure for CF.”

Hamlin began donating money from his race winnings to the Cystic Fibrosis Foundation when he started racing go karts as a child.

Pediatric Research Office

Medical Director: Henry Rozycki, MD, Ph: 8-9965 Email: hrozycki@mcvh-vcu.edu Administrator: Jaime Petrasek Ph: 6-0212 Email: petrasekjl@vcu.edu Grants Specialist: Calcine Harris Ph: 6-1569 Email: charris32@vcu.edu Research Coordinators: Debbie Bailey RN Ph: 6-2632 Email: dabailey@vcu.edu Matt Ward Ph: 5-3039 Email: wardmt@vcu.edu

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New Grants & Contracts AwardedNew Grants & Contracts AwardedNew Grants & Contracts Awarded

Francis, Gary L. Pediatrics A Trial Investigating the Efficacy and Safety of Insulin Degludec in Children and Adolescents with Type 1 Diabetes Mellitus

Novo Nordisk Pharmaceuticals, Inc

Schmidt, Howard J. Pediatrics Therapeutics Development Center – 2012 Cystic Fibrosis Foundation Godder, Kamar

Pediatrics An Open-label, Extension Study to Assess the Long- Term Safety of Twice Daily Oxycodone Hydrochloride Controlled-release Tablets in Opioid Experienced Children Who Completed the OTR3001 Study

Purdue Pharma LP

Irani, Anne-Marie Pediatrics A Prospective, Randomized, Double-Blind Controlled Study to Evalu-ate the Nutritional Safety (Growth) and Hypoallergenicity of an Amino Acid Based Formula With Prebiotics and Probiotics in Infants Diagnosed With Cow Milk Allergy, With or Without Other Food Aller-gies

SHS International Ltd./ Nutricia North America

Irani, Anne-Marie Pediatrics Phase IV Study to Evaluate the Persistency of Asthma With or Without Xolair After Long Term Therapy

Genentech, Inc.

Francis, Gary L. Pediatrics A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Saxagliptin (BMS-477118) in Combination with Metformin IR or Metformin XR in Pediatric Patients with Type 2 Diabetes who have Inadequate Glycemic Control on Met-formin Alone

Bristol-Myers Squibb Company

Rozycki, Henry J. Pediatrics TLR4 Function in Premature Lung Cells

Jeffress Memorial Trust Rubin, Bruce K. Pediatrics An Observational Study to Evaluate the Relationship of Nasal Mucus

Properties and Symptoms in Acute Rhinosinusitis

McNeil Consumer Healthcare

Rubin, Bruce K. Pediatrics The Effect of Humidification on Mucus Rheology Fisher & Paykel Healthcare, Ltd

Elsea, Sarah H. Pediatrics A cell culture model to detect drug-induced expression of RA11 Smith-Magenis Research Founda-tion

Hendricks-Munoz, Pediatrics Reducing Health Disparities Among Minority and Underserved Chil-dren (R21)

National Institute of Child Health and Development/NIH

Kumar, Santhosh Pediatrics A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Paral-lel-Group Study Evaluating the Efficacy and Safety of Grass (Phleum Pratense) Sublingual Tablet (SCH 697243) in Subjects Between 5 and 65 Years of Age, With a History of Grass Pollen-Induced Rhinocon-junctivitis, With or Without Asthma (Phase 3; Protocol No. P08067)

Merck & Co.

Gupta, Anshu Pediatrics Role of advanced glycation end products (AGEs) in polycystic ovary syndrome (PCOS)

AD Williams.

Congratulations to the following Principal Investigators who were awarded grants and contracts in the first half of 2012!

Anyone with a grant application, or an inquiry or an idea is encouraged to call the Pediatric Research Office. We can help with regulatory document review and preparation (OSP, IRB, WIRB, IACUC), with budget preparation, with get-ting signatures or with finding resources within the University to help you accomplish your research goals. Call or email Jaime Petrasek or Henry Rozycki.

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Publications First Half 2012Publications First Half 2012Publications First Half 2012

Williams SR, Zies D, Mullegama SV, Grotewiel MS, Elsea SH. Smith-Magenis Syndrome Results in Disruption of CLOCK Gene Transcription and Reveals an Integral Role for RAI1 in the Maintenance of Circadian Rhythmicity. Am J Hum Genet. 2012; 90(6):941-9.

Chung BH, Mullegama S, Marshall CR, Lionel AC, Weksberg R, Dupuis L, Brick L, Li C, Scherer SW, Aradhya S, Stavro-poulos DJ, Elsea SH, Mendoza-Londono R Severe intellectual disability and autistic features associated with microdupli-cation 2q23.1.Eur J Hum Genet. 2012;20:398-403.

Adjustable passive stiffness in mouse bladder: regulated by Rho kinase and elevated following partial bladder outlet obstruc-tion.Speich JE, Southern JB, Henderson S, Wilson CW, Klausner AP, Ratz PH. Am J Physiol Renal Physiol. 2012;302:F967-76.

Jönsson F, Mancardi DA, Zhao W, et al. Human FcγRIIA induces anaphylactic and allergic reactions.

Throckmorton AL, Sciolino MG, Downs EA, Saxman RS, López-Isaza S,Moskowitz WB. Controlled pitch-adjustment of impeller blades for an intravascular blood pump. ASAIO J. 2012;58:382-9.

Furlan, A.J., Reisman, M., Massaro, J., Mauri, L., Adams, H., Albers, G.W., Felberg, R., Herrmann, H., Kar, S., Landzberg, M.,Raizner, A., and Wechsler, L., for the CLOSURE I Investigators*: Closure or Medical Therapy for Cryptogenic Stroke with Patent Foramen Ovale. N Engl J Med 2012;366:991-999. *W.B. Moskowitz

Hart, R., Gibson, D, Bean, M.K., & Fisher, R. Alcohol history differentiates cognitive functioning among patients undergo-ing evaluation for liver transplantation. International Journal of Clinical Medicine,2012; 3:125-131.

Le TN, Nestler JE, Strauss JF 3rd, Wickham EP 3rd Sex hormone-binding globulin and type 2 diabetes mellitus.Trends En-docrinol Metab. 2012;23:32-40.

Bean MK, Miller S, Mazzeo SE, Fries EA.Social cognitive factors associated with physical activity in elementary school girls.Am J Health Behav. 2012;36:265-74.

Huddle LN, Fuller C, Powell T, Hiemenga JA, Yan J, Deuell B, Lyders EM, Bodurtha JN, Papenhausen PR, Jackson-Cook CK, Pandya A, Jaworski M, Tye GW, Ritter AM.Intraventricular twin fetuses in fetu.J Neurosurg Pediatr. 2012;9:17-23.

Aygun B, Wruck LM, Schultz WH, Mueller BU, Brown C, Luchtman-Jones L, Jackson S, Iyer R, Rogers ZR, Sarnaik S, Thompson AA, Gauger C, Helms RW, Ware RE; TCD With Transfusions Changing to Hydroxyurea (TWiTCH) Trial Investigators*. Chronic transfusion practices for prevention of primary stroke in children with sickle cell anemia and ab-normal TCD velocities.Am J Hematol. 2012;87:428-30. *K Godder

Miller ST, Kim HY, Weiner D, Wager CG, Gallagher D, Styles L, Dampier CD; Investigators of the Sickle Cell Disease Clinical Research Network (SCDCRN)*.Inpatient management of sickle cell pain: a 'snapshot' of current practice.Am J Hematol. 2012;87:333-6. *K Godder

Gilman AL, Schultz KR, Goldman FD, Sale GE, Krailo MD, Chen Z, Langholz B, Jacobsohn DA, Chan KW, Ryan RE, Kel-

lick M, Neudorf SM, Godder K, Sandler ES, Sahdev I, Grupp SA, Sanders JE, Wall DA. Randomized trial of hydroxy-chloroquine for newly diagnosed chronic graft-versus-host disease in children: a Children's Oncology Group study. Biol Blood Marrow Transplant. 2012;18:84-91.

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Nigro G, Adler SP, Parruti G, Anceschi MM, Coclite E, Pezone I, Di Renzo GC. Immunoglobulin therapy of fetal cytomega-

lovirus infection occurring in the first half of pregnancy--a case-control study of the outcome in children. J Infect Dis. 2012 ;205:215-27.

Saiman L, Mayer-Hamblett N, Anstead M, Lands LC, Kloster M, Goss CH, Rose LM, Burns JL, Marshall BC, Ratjen F; the AZ0004 Macrolide Study Team. Open-label, follow-on study of azithromycin in pediatric patients with CF uninfected with Pseudomonas aeruginosa. Pediatr Pulmonol. 2012;47:641-648.

Kuchta AL, Parikh H, Zhu Y, Kellogg GE, Parris DS, McVoy MA. Structural modelling and mutagenesis of human cy-tomegalovirus alkaline nuclease UL98. J Gen Virol. 2012;93:130-8.

Bailey SM, Hendricks-Muñoz KD, Mally P Splanchnic-cerebral oxygenation ratio as a marker of preterm infant blood transfusion needs. Transfusion. 2012;52:252-60.

Chapman AK, Lehmann CU, Donohue PK, Aucott SW. Implementation of computerized provider order entry in a neonatal intensive care unit: Impact on admission workflow. Int J Med Inform. 2012;81:291-5.

Chapman AK, Aucott SW, Milstone AM. Safety of chlorhexidine gluconate used for skin antisepsis in the preterm infant. J Perinatol. 2012;32:4-9.

Lavergne V, Nolin TD, Hoffman RS, Roberts D, Gosselin S, Goldfarb DS, Kielstein JT, Mactier R, Maclaren R, Mowry JB, Bunchman TE, Juurlink D, Megarbane B, Anseeuw K, Winchester JF, Dargan PI, Liu KD, Hoegberg LC, Li Y, Calello DP, Burdmann EA, Yates C, Laliberté M, Decker BS, Mello-Da-Silva CA, Lavonas E, Ghannoum M. The EXTRIP (EXtracorporeal TReatments In Poisoning) workgroup: guideline methodology. Clin Toxicol (Phila). 2012;50:403-13.

Bunchman TE.Biomarkers for acute kidney injury: is the serum creatinine worthless? Pediatr Crit Care Med. 2012;13:119-20.

Rubin BK, Pohanka V. Beyond the guidelines: fatal and near-fatal asthma. Paediatr Respir Rev. 2012;13:106-11.

De Blasio F, Dicpinigaitis PV, Rubin BK, De Danieli G, Lanata L, Zanasi A. An observational study on cough in children: epidemiology, impact on quality of sleep and treatment outcome. Cough. 2012;8:1.

Robinson P, Schechter MS, Sly PD, Winfield K, Smith J, Brennan S, Shinkai M, Henke MO, Rubin BK. Clarithromycin therapy for patients with cystic fibrosis: a randomized controlled trial. Pediatr Pulmonol. 2012;47:551-7.

Publications First Half 2012Publications First Half 2012Publications First Half 2012

Papers in print or online before publication will be included in the next newsletter. If any publica-tions were missed, please email Henry Rozycki and we will include them in the next newsletter.

Congratulations to all the authors of these 27 publications!

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CHOR@VCU on the move!CHOR@VCU on the move!CHOR@VCU on the move! Presentations to National & International MeetingsPresentations to National & International MeetingsPresentations to National & International Meetings

Houston TX - Society of Critical Care Medicine Conference Feb 4-8, 2012

Azadeh Fayazi, Gary Francis, Mark Marinello, Sue Sreedhar, Leroy Thacker, Lorin Bachmann. Is Vitamin D Deficiency As-sociated with Critical Illness in Children?

New Orlean LA - Annual Winter Meeting of the Society for Urodynamics and Female Urology (SUFU). Feb 28- Mar 3, 2012

Byrne MD, King AB, Speich JE, Klausner AP, Ratz PH. A new method for objective analysis of detrusor rhythmic contrac-tion. Southern JB, Frazier JR, Minor AS, Byrne MD, King, AB, Speich JE, Klausner AP, Ratz PH. Length-dependent regulation of detrusor smooth muscle myosin light chain phosphorylation and tone during bladder filling

Speich JE, Almasri AM, Wilson CW, Southern JB, Klausner AP, King AB, Byrne MD, Ratz PH. Length adaptation via autonomous contraction (AC) in rabbit detrusor and volume adaptation in mouse bladder.

Klausner AP, King AB, Rogman A, Speich JE, Byrne MD, Ratz PH. Prostanoid profiling using ion-spray tandem mass spec-trophotometry: a potential new modality for overactive bladder diagnosis and sub-typing.

Tenerife, Spain — International Course in Pediatric Pulmonology, Mar 22-26, 2012

Rubin, BK. [1] Secretory hyperresponsiveness and persistent atelectasis [2] Interstitial ling disease in children, [3] Diagnosis and management of non-CF bronchiectasis in children.

San Diego CA - Experimental Biology. Apr 21-25, 2012

Ratz PH, Barbee RW, Miner AS, Reynolds PS, Contaifer D. Reduced Vascular Smooth Muscle (VSM) Sensitivity to Con-traction in ex vivo Hemorrhaged Rabbit Epigastric Artery (EA).

Boston MA - Pediatric Academic Societies. April 28-May 1, 2012

Wickham, EP, Jones, RM, Sylvester, C., Evans, R., Bean, MK, Ning, Y. Comparison of 48- and 24-hour Dietary Recall Methods in Predicting Changes in Body Mass Among Overweight Adolescents Enrolled in a Multidisciplinary Weight Man-agement Program. Bean, MK., Biskobing, DM., Francis, GL, Wickham, EW. Motivational Interviewing in Healthcare: Results of a Brief Train-ing in Endocrinology. Chapman A, Aucott S, Gilmore M, Advani S, Clarke W, Milstone A. Safety of chlorhexidine gluconate (CHG) use in pre-term infants in the first 2 weeks of life. Carr C, Wijesooriya NR, Francis G, Gennings C, Wickham E. Urinary Phthalate Levels and Obesity in U.S. Children: Are They Related? Astoria M, Karam S, Magill C, Rozycki HJ, Moores R. Cardiac-Specific Troponin as a Predictor of Outcome in Neonatal ECMO. Jayaram A, Feldman M, Moores R, Brock E. Performance of Basic Neonatal Resuscitation Skills by Pediatric Residents. Fox JR, Rozycki HJ. Should Premature Infants Who Receive Prophylactic Surfactant Be Routinely Evaluated and Treated for Sepsis? Bunchman TE. Current State of Extracorporeal Renal Replacement Therapy Bunchman TE. Devices To Support Neonatal AKI Injury.

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San Francisco CA – American Thoracic Society International Meeting. May 18 – 23, 2012 Tanabe T, Shimokawaji T, Kanoh S, Rubin BK. IL-33 Increases IL-8 Secretion From Human Airway Goblet Cells Mikita K, Kanoh S, Tanabe T, Kawano S, Hara Y, Fujikura Y, Maeda T, Kawana A, Rubin BK, Tokorozawa JP. Macrolides Inhibit Interleukin-13-Induced Goblet Cell Hyperplasia In Human Airway Cells Kanoh S, Tanabe T, Rubin BK. Dapsone Attenuates IL-13-Induced MUC5AC Expression And Goblet Cell Differ-entiation In Human Bronchial Epithelial Cells Tanabe T, Shimokawaji T, Kanoh S, Rubin BK. Secretory Phospholipases A2 Are Produced In Ciliated Airway Cells And Increase Goblet Cell MUC5AC Rozycki HJ, Brock A, Webb-Parker S, Tanabe T. Increased CXCL2 Production From Mouse Type II Alveolar Epithelial Cells In Response To The Alarmin HMGB1 Rubin BK. 1) Professor’s Rounds—Plastic Bronchitis. 2) Symposium—Pathways to drug development.

Atlanta GA – American Urological Association, May 19-23, 2012

Ratz PH, Klausner AP, Speich JE. How might bladder filling phase contractile activity affect urgency and the power of voiding contraction?

San Francisco CA – American Society for Artificial Internal Organs. June 14-16, 2012 Downs EA, Moskowitz WB, Throckmorton AL. Intravascular Mechanical Cavopulmonary Assistance of 3-D Pa-tient-Specific Fontan Configurations. Throckmorton AL, Sciolino MG, Downs ES, Lopez-Isaza S, Moskowitz WB. Controlled Pitch-Adjustment of Im-peller Blades for an Intravascular Blood Pump

Bangkok Thailand —International Congress of Pediatric Pulmonology (CIPP XI) June 28—July 3, 2012.

Rubin BK. [1] Management of life threatening asthma, [2] New aerosol drugs and devices, [3] How to get your paper published (workshop). [4] Pediatrics is magical

CHOR@VCU on the move!CHOR@VCU on the move!CHOR@VCU on the move! Presentations to National MeetingsPresentations to National MeetingsPresentations to National Meetings

CHOR@VCU on the move!CHOR@VCU on the move!CHOR@VCU on the move! Presentations to National & International MeetingsPresentations to National & International MeetingsPresentations to National & International Meetings

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Children’s Hospital of Richmond at VCUChildren’s Hospital of Richmond at VCUChildren’s Hospital of Richmond at VCU Current and Upcoming Clinical TrialsCurrent and Upcoming Clinical TrialsCurrent and Upcoming Clinical Trials

There are currently more than eight clinical trials actively enrolling patients through the Pediatric Re-search Office. Several more not listed here are being done in the CRSU or within specific labs and clini-cal divisions. There is a web page dedicated to clinical research trials on the Department web site at http://www.pediatrics.vcu.edu/research/patients.html. We plan to improve it by highlighting actively re-cruiting trials. Even more exciting news is that there are 9 clinical trials in the pipeline and expected to begin enrolling patients beginning this fall.

Clinical trials can be investigator-initiated or come from sponsors. If any help is required regarding: ♦ Feasibility, e.g. are there enough subjects? ♦ Resource identification ♦ Budget construction ♦ Regulatory requirements and documentation ♦ Protocol development ♦ Study implementation ♦ Finances ♦ Close-out ♦ Others Please contact Jaime Petrasek or Henry Rozycki

Gary L. Francis Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Saxagliptin as Monotherapy in Pediatric Patients With Type 2 Diabetes

Gary L. Francis Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus

Gary L. Francis A trial investigating the efficacy and safety of insulin degludec in children and adolescents with type 1 diabetes mellitus

Gary L. Francis Type 1 Diabetes Trial Net

Kamar Godder Safety of Twice Daily Oxycodone Hydrochloride Controlled-release Tab-lets in Children With Moderate to Severe Malignant and/ or Nonmalig-nant Pain Requiring Opioids

Linda Meloy Preterm Infant Growth

Suzanne Lavoie An Open-Label,Multicenter, Multiple-Dose Pharmacokinetic and 48-week safety and Efficacy Trial of Maraviroc in Combination with Optimized Background Therapy for the treatment of Antiretroviral-experienced CCR5-Tropic HIV-1 Infected Children 2-18 years of age