Report

2012 · 2015

GETHI

LETTER FROM THE PRESIDENT 4

ABOUT GETHI 6 Mission and vision 8 Objectives 9

STRUCTURE Board of Directors 10 Work groups 11

ACTIVITY REPORT 12 Research 13 •Clinicaltrials 13 •Tumourregistry 14 Scientificdissemination 16 •Magazines 16 •Guidesandmanuals 16 •Scientificarticles 17 •Communicationatconferences 18 •PosterSessions 18 Training 19 •Conferencesandmeetings 19 •Grants 20 •Apps 21

COMMUNICATION 22 Website 22 Socialnetworks 22 GETHIinthemedia 23

Index Report2012 · 2015

GETHI

Report2012 · 2015

GETHILETTER FROM THE PRESIDENT

04 05

Through Directive 24/2011, the European CommissionurgedthememberstatesoftheEuropeanUniontodevelopReferenceNetworks,with the aimof improving the careand quality of life of patients with rare diseases and, byextension, those with rare tumours. The first objectiveoftheReferenceNetworksistoconnectthemosthighlyqualifiedprofessionalswithSpecialisedReferenceCentres,thereby achieving a concentrationof knowledge in thoseareaswhereresourcesarescarce.

This idea inspiredthecreationoftheSpanishTaskForceGroupforOrphanandInfrequentTumours(GETHI),whichwasfoundedinFebruary2012thankstothehelpandsupportoftheSpanishSocietyofMedicalOncology(SEOM).Sincethen,ourgrouphaspromotedtheparticipationandcollaborationofprofessionalsworkinginclinicalpractice,epidemiologyandbasiccancerresearch,thuscreatinganetworktodriveclinicalresearch,toshareinformationandexperiencesthroughtheregistriesofraretumoursand,aboveall,tobeabletoofferourpatientsbettercare.

It is for this reason that, as current President of GETHI, I would first like toacknowledgetheworkcarriedoutbythepreviousteam,andespeciallyDr.EnriqueGrandeasmotivatorof thegroup;hewastheonewhoset itupandgotusonboardwiththeideathatitisnecessaryforustoknoweachotherandtoshareourknowledge.

During these three yearsof existence,wehave focusedourworkon increasingthenumberofspecialistswhosemedicalpracticeworkisrelatedtosometypeofinfrequentneoplasm.Therefore,ourinitialobjectivewastoestablishnetworksofexpertspecialistsatanational level,whichwouldprovideuswith fasterwaysofcommunicatingwitheachotherandwouldbreakdownthegeographical,knowledgeandexperiencebarriers,which isthemainconcernofthepatientwhohasbeendiagnosedwithararecancerandgoesinhopetotheironcologist.

Atthesametime,scientificresearchintothesetypesoftumoursandthetrainingofprofessionalsinterestedinthisareahasbeen,andcontinuestobe,theGroup’smainpriority.

Throughopinionforums,workgroups,publicationsandprofessionalmeetingswewanttorespondtotherealityofOncologyofOrphanandInfrequentTumoursandtomeetthenewchallengesthatemergeinthetreatmentandresearchofthesame,whichwillresultinbetterpatientcare.

Wehavealreadycreatedvariousregistriesofraretumourfamilies–thyroid,bronchialcarcinoid, intestinal neuroendocrine– and registries of other families are in theprojectphase.Weareworkingcloselywithothercooperativegroupswhich,fromanotherperspective, have similar registries, such as the SpanishNeuroendocrineTumourGroup(GETNE)andtheSpanishSarcomaResearchGroup(GEIS).Let’snotforgetthat,intheareaofresearch,importantclinicaltrialshavealreadybeenconducted which clearly demonstrate a before and after in the treatment ofinfrequenttumours.

Ouractivityisdefinedbythefuture.Apatientdiagnosedwithaninfrequenttumourshouldnothavetoexperiencethesenseofisolationcausedwheninformedoftherarityoftheirdisease.Theoncologistattendingthepatientmustconveythat,inthecontextofanextensivenetworkofexpertspecialiststherareorinfrequenttumourceasestobeso.

GETHIhasgreatfutureprospects,bothintermsoftheprogressiveexpansionofournetworkofcontactsandtheinterestgeneratedinestablishingepidemiologicaland clinical research projects. I therefore encourage all interestedmembers andspecialists to participate actively in GETHI in order to achieve the objectivesproposedatthebeginningofthisletter.

Ramón de las Peñas PresidentofGETHI

Report2012 · 2015

GETHIABOUT GETHI

06 07

Raretumourspresentachallengeinthedailypracticeofmedicalprofessionalsfromadiagnosticandtherapeuticpointofview.Despitebeingconsideredthethirdorfourthmostcommontumourintermsofincidence,andthefactthattheyaccountforaround20%ofallsolidtumours,theseneoplasmsarediagnosedseparately in fewerthan5casesper100,000peopleeachyear.Thismeansthatprofessionalsrarelycomeacrossthesediseasesintheirdailyworkandthatpatientswhosufferfromthemoftenfeelisolated.

The Spanish Task Force Group for Orphan and Infrequent Tumours(GETHI)wasestablishedon9February2012withtheaimofpromotingbasic,translationalandclinicalresearchintoraretumoursandofcreatinganetworktosupporttheworkofoncologyprofessionalsinthisfield.

GETHIcurrentlyhas132members,themajorityofwhomaremedicaloncologyspecialists,from78Spanishhospitalsandresearchcentres.

“Research into rare tumours is not only a moral duty, but also a unique source of knowledge that will benefit

the whole of society”

GETHImembersbyautonomouscommunity

Report2012 · 2015

GETHIABOUT GETHI

08 09

ObjectivesTo create a national network of professionals that will enable medicaloncologyprofessionals to access the information theyneed about infrequentneoplasms in apersonal, simple and completewaywithouthaving to consultforeignregistries.

Toserveasabridgeandtoprovidesupportinthecollaborationwiththevariouscooperativegroupsworkinginthefieldofneoplasmsinwhichtherearerareorinfrequentvariants,promotingthemutualenrichmentofthegroupsforthebenefitofpatients.

To quantify the impact of rare tumours in Spain, using population-basedregistriesthatofferreal,qualitydataonincidenceinSpain.

Totrainspecialistsinraretumours,whocanpromotebasic,translationalandclinicalresearchinthefuture.

Todrawuptreatmentguidelinesandclinicalpracticeprotocolsthatenableprofessionalstooffergreaterqualityofcaretopatients.

Tosupportresearchandaccesstonewtreatments,as theprincipal lobbygroupformedbyexpertsinraretumoursinSpain.

Topromotestrategicpartnershipswithscientificinstitutionsandcompaniesfocused on studying and raising awareness about rare diseases, in order tocombineoureffortsinthefightagainstthesepathologies.

MissionandvisionGETHIseekstoachieveaqualitativeleapintheapproachandresearchintoorphanandinfrequenttumours.Throughthepromotionoftrainingactivities,conducting own and collaborative research and the creation of networks ofprofessionalsinraretumours,GETHIaimstocontributedirectlytoenriching scientificknowledgeandtotheexpertiseofourprofessionalstobeabletooffer greaterqualitycaretopatients.

We aspire to be a scientificgroupofreferenceforinfrequentneoplasms,bothinSpainandabroad;agroupthatcanprovideprofessionalswiththesupport,resourcesandtoolstheyneed,aswellasinvolvetheminthiscommonprojectaimedatmakingraretumoursincreasinglylessunknown.

Wearecertainthattheeffortsofallofuswhostrivetogaingroundonthesetumourswillonedaychangethehistoryofcancer.

Report2012 · 2015

GETHISTRUCTURE

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BoardofDirectors2015-2018

Dr. Juan Antonio VirizuelaH.U.VirgendeMacarena

Member

Dr. Ramón de las PeñasConsorcioHospitalarioProvincialdeCastellón

President

Dr. Martín LázaroComplexoHospitalarioUniversitariodeVigo

Vice-President

Dra. Núria RodríguezHospitalUniversitarioLaPaz

Secretary

Dr. Jesús García-DonasCentroIntegralOncológicoClaraCampal

Treasurer

Dr. Enrique GrandeH.U.RamónyCajal

Member

Dr. Jaume CapdevilaH.U.Valld´Hebrón

Member

Dr. Carlos LópezH.U.MarquésdeValdecilla

Member

Dr. Federico LongoHospitalRamónyCajal

Member

Workgroups

TUMOUR

Pheochromocytoma/Paraganglioma

AdrenocorticalCarcinoma

Non-GliomaBrainTumours

Non-MelanomaSkinTumours

Thymoma

ThyroidTumours

EyeTumours

RareHeadandNeckTumours

Children’sTumoursinAdults(Wilms,Medulloblastoma,PNET)

RareSarcomas

Sarcomas/NeuroendocrineTumours

TumoursofUnknownOrigin

Othertumours

GETHI Activities

19 % 19 %11 % 5 % 3 % 5 %8 %16 % 14 %

Scientificarticles Registryoftumours Apps

Communicationstoconferences Meetings and conferences Journals

Clinicaltrials Grants Guidesandmanuals

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PatientswithinfrequenttumoursarethemainfocusofGETHI’sactivity.Research,scientific dissemination and training of professionals are the key instrumentsforcontributing to improving theoutlookandcareofpatientsand thereforeconstitutethemainpillarsonwhichtheGroup’sworkisfounded.

Sinceitscreationin2012,GETHIhasimplementedover25researchandtrainingprojectsandhashelpedincreaseknowledgeaboutthesetypesoftumoursthroughthecreationofthejournalUncommon Oncology,thepublicationofvariousguidesandmanuals,andbywritingordirectlycontributingtoseveralscientificarticles

Areasofactivity

38 %

30 %

32 %

PUBLICA

TION

S

RESEA

RCH

TRA

ININ

G

ResearchCLINICAL TRIALS

Greko I: Ketoconazole in the treatment ofmetastatic ovariangranulosa-cellcarcinoma

The aim of this trial was to study the benefits of Ketoconazole inthe treatment of ovarian granulosa-cell carcinomas, in order to beable tooffer an alternative therapywith lower toxicity. Greco I isthefirstattemptattreatingovariangranulosa-cellcancerwithasolidmolecularrationale.

Greco II:Orteronel inmetastaticoradvancednon-respectableovariangranulosa-celltumours

Themainobjectiveofthistrialistoevaluatetheclinicalbenefitrateand to determine the toxicity profile of Orteronel in metastaticovariangranulosa-celltumours.

Molecularalterationsinoligodendroglialtumours

This trial involves studying the underlying molecular changes inoligodendroglialbraintumours,inordertounderstandthemechanismsof evolution between these types of tumours and to find effectivetherapeutictreatmenttargetsforthisorphanpathology.

GrekoIII:Enzalutamideinovariangranulosa-celltumours

TheobjectiveofGrekoIIIistogatherscientificevidenceofthepotentialofEnzalutamideinthetreatmentofovariangranulosa-cellcancer.Thestudyisthereforefocusedonevaluatingtheoverallresponserateofpatients,establishingtheclinicalbenefitanddeterminingthetoxicitylevelofthedrug.

PI: Jesús García Donas, CIOCC

PI: Jesús García Donas, CIOCC

PI: Jesús García Donas, CIOCC

PI: María Ángeles Vaz, H.U. Ramón y Cajal

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ResearchThe Pembro-rare trial: A multi-centre phase II study ofPembrolizumabinpatientswithrarecancers

ThisisthefirstclinicaltrialinPhaseIIofthecompoundPembrolizumabinraretumours,wheremolecularresearchiscarriedoutinordertoidentifywhichcasesaremostlikelytobenefitfromthistherapy.

Pembrolizumab maintenance after first-line chemotherapytreatmentforCancerofUnknownPrimary(CUP),aphaseIItrialconductedbyGETHI

This clinical trial is aimed at studying Pembrolizumab maintenanceafterthefirstlineofchemotherapytreatmentforCancerofUnknownPrimary(CUP).ItalsoaimstopredicttoxicityandtoincreasethetotalresponserateinpatientsbeingtreatedwithPembrolizumab

TUMOUR REGISTRY

Registryofbronchialcarcinoids

Thisregistryfavourstheexchangeofexperienceswiththerapeuticsolutionsusedbyprofessionalsinbronchialcarcinoids,inordertopromotetheestablishmentofconsensusguidelinesaimedatstandardisingtreatments.

MedullaryThyroidCarcinoma

Atnationallevel,thisregistrywascreatedtoincludethemaximumnumberofcasesofmedullarythyroidcarcinoma,regardlessoftheirstage,thetreatmentsusedorthemolecular-geneticstateofthetumour.

Differentiated/undifferentiatedradioiodine-refractorythyroidcarcinoma

Thisnationwideregistrycanincludeallcasesofdifferentiated/undifferentiatedthyroidcarcinomaswhichhavedemonstratedrefractorinesstotreatmentwithradioactive isotope

GETHI-TOD-DATABASE

Thisproject,whichiscurrently inthedevelopmentstage, isaimedatcreatinga registry of tumours of unknown origin at a national level, and centralisedwithin theGETHIgroup. Theobjective is to includeclinical andpathologicalcharacteristicsinordertoacquireabetterknowledgeofdescriptivestatisticsandoftheprognosisandtreatmentoftumoursoforigin.

PI: Federico Longo, H.U. Ramón y Cajal

PI: Xabier Mielgo, H. Fundación de Alcorcón

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ScientificdisseminationJOURNALS

Uncommon OncologyThe journalUncommon Oncology, sponsored byGETHI,was presented at the“OncologyDiscussionForum2012”asadisseminationtoolforraretumourswithinfrequentevolution.

EditedbyDr.JavierMartínezTruferoandDr.AntonioAntónTorres,itincludesreviewarticlesandclinicalcasestudiesonthetreatmentappliedanditsresults.Atotalofsevenissuesof Uncommon Oncologyhavebeenpublishedtodate.

BOOKSANDMANUALS

Guidelines for the therapeutic management of Caprelsa in advancedmedullarythyroidcancerTheaimofthisguideistosummarisetheinformationpublishedontheadverseeffectsassociatedwithVandetanibtherapyinadvancedmedullarythyroidcancer,andtoprovidedoctorswithpracticalguidelinesregardingeducation,monitoringandmanagementofthetoxicitiesinducedinpatientsundergoingthistreatment.

TreatiseonRareTumoursThe numberof rare tumours diagnosed is expected to continue to increaseover thecomingyears. It is thereforeessential tohaveaspecificknowledgeofthephysiologyoftheorgansinwhichtheyoccur.Thistreatisereflectstheneedtoincludeinasingleworktheparticularitiesofthetreatmentofthemaininfrequentneoplasms.

GETHImonographofParaneoplasticSyndromesParaneoplastic Syndromes occur before or during the development of theneoplasm. When they appear during the development stage, both clinicalmonitoringandthedetectionofproteinsorantibodiescanbeusedastumourmarkers for the course of the primary neoplasm. In thismonograph,GETHIpromotesareviewoftheseprocessescarriedoutbyspecialistsinvolvedinthediagnosisandmonitoringofthesame.

SCIENTIFIC ARTICLES

•Garcia-Donas J, Hurtado A, García-Casado Z, Albareda J, López-Guerrero JA, Alemany I, Grande E, Camara JC, Hernando S. CytochromeP17InhibitionWithKetoconazoleAsTreatmentforAdvancedGranulosaCellOvarianTumor. J Clin Oncol. 2013 Apr 1; 31(10):e165-6

•Grande E, Capdevila J, Díez JJ, Longo F, Carrato A. Asignificantresponsetosunitinibinapatientwithanaplasticthyroidcarcinoma.J Res Med Sci. 2013 Jul; 18(7):623-5

•Grande E, Kreissl MC, Filetti S, Newbold K, Reinisch W, Robert C, Schlumberger M, Tolstrup LK, Zamorano JL, Capdevila J. Vandetanibinadvancedmedullarythyroidcancer:reviewofadverseeventmanagementstrategies. Adv Ther. 2013 Nov; 30(11):945-66

•Gatta G, Mallone S, van der Zwan JM, Trama A, Siesling S, Capocaccia R; EUROCARE Working Group. CancerprevalenceestimatesinEuropeatthebeginningof2000. Ann Oncol. 2013 Jun; 24(6):1660-6

•Prieto I, Pérez de la Fuente T, Medina S, Castelo B, Sobrino B, Fortes J, Esteban D, Cassinello F, Jover R, Rodríguez N. Merkel cell carcinoma: an algorithm formultidisciplinarymanagementanddecision-making. Critical Reviews in Oncology/Hematology (in press)

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ScientificdisseminationCOMMUNICATION AT CONFERENCES

ASCO 2014 (Abstract #130461)• García-Donas, J. Ketoconazole as inhibitor of the enzyme CYP17 in locally advanced

or disseminated granulosa cell tumors of the ovary. The GreKo I study (gethi 11-03).

POSTER SESSIONS

• ASCO 2014Hurtado Nuño, A. Open-labelphaseIIclinicaltrialofOrteronel(TAK-700)inmetastaticoradvancednonresectablegranulosacellovariantumors:TheGreKoIIstudy. Citation: J Clin Oncol 32:5s, 2014 (suppl; abstr TPS5626)

• ESMO 2014 (Abstract #7255)

Grande, E. SafetyandefficacyofVandetanibassystemictreatmentforpatientswithadvancedandprogressiveMedullaryThyroidCancer(MTC).

• SEOM XIV National Medical Oncology Meeting 2014 (Poster Session 188-PD).

Hurtado Nuño, A. OpenphaseIIstudyofKetoconazoleasinhibitoroftheenzymeCYP17 in locally advancedor disseminated granulosa-cell tumours of the ovary.GreKostudy.

TrainingCONFERENCES AND MEETINGS

MedullaryThyroidCancerExpertMeeting

With the aim of discussing and establishing an international multidisciplinaryconsensusonthetreatmentofpatientswithadvancedmedullarythyroidcancer,in October 2012 GETHI organised the“MedullaryThyroid Cancer ExpertMeeting”,whichbroughttogetherspecialistsfromdifferentdisciplines.

SymposiumofRareTumours:Practicalconcepts

Althoughmultidisciplinary teams are usual in Spain’s hospitals, this situation istrulyexceptionalwhenitcomestoraretumours.

The symposium involved the creation of a meeting point for professionalsinterestedinorphanandinfrequenttumours.Theflowofinformationgeneratedthroughtheexchangeofexperienceshas,withoutadoubt,contributedtounifyingthetreatmentcriteriafortheseneoplasms.

GETHIAfterASCO2014

EverymedicaloncologyprofessionalisawareoftheimportanceofthemeetingoftheAmericanSocietyofClinicalOncology(ASCO),whichannuallypresentsthemainmilestonesincancerresearch.

In2014,GETHIorganisedthisseminartoupdateknowledgeonthenewdevelopmentspresentedatASCOwithregardtoraretumours.Theseminaraddressedraretumourspecialitiesandover200abstractswerereceived.

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TrainingRESEARCH GRANTS

IncollaborationwiththeFundaciónConsorcioHospitalProvincialdeCastellón,in2013theGroupestablishedtheirfirstgrantprogramme“GETHI–RamóndelasPeñasdeAyudaalaInvestigaciónenTumoursHuérfanoseInfrecuentes”.

Thegrantprogramme,asecondeditionofwhichhasbeenconvenedfor2015,wascreatedaspartof theGroup’s strategy topromote thedevelopmentofresearch projects that benefit the progress of clinical therapy and increaseknowledgeaboutthesetumours.

INTERVIEW

Q.Ingeneralterms,whatistheaimandraisond’êtreofyourproject?Westudywhichmechanismsareinvolvedintheunderlyingmolecularchangesinoligodendroglialtumoursandwelookforavenuesforthedevelopmentofnewdrugs.

Q. HowhastheGETHIgranthelpedthedevelopmentoftheproject?Researchfundingiskeytoscientificadvancesinthesetypesoftumours.WithoutGETHI’shelp,astudylikethiswouldhavebeenverydifficulttocarryout.

Q.Inyouropinion,whatrolecanGETHIplayinSpanishoncology? TheGroupobviouslyplaysafundamentalroleinthedevelopmentofresearchprojects,inpromotingtrainingandinthedisseminationofinformationaboutraretumours.Ithinkthatithasestablishedaveryimportantmeetingpointforthedifferentspecialistswhoworkinthisfield.

APPS

MTC

Medullarythyroidcancerisresponsiblefor13.4%ofdeathscausedbythistypeoftumour.Forthisreason,GETHIbelievesitessentialtoprovidethesetreatmentguidelinesinappformat,explainingthegeneralguidelinestofollowwhenmanagingMTCandusingVandetanibasatreatment.

TreatiseonRareTumours

The consensus document drawn up by the Group served as a basis for thecreationofthisapp,whichoffersinaquickandintuitivewaythekeysforbetterunderstandingtheparticularitiesinvolvedintreatingraretumours.

GIST

Thisapplicationoffersaprotocol for thetreatmentofgastrointestinal stromaltumours(GIST).Thesetumoursarerarecancersthatoriginate fromtheCajalcells,whicharethecellsresponsible for initiatingtherhythmicmovementthatpushesfoodthroughthedigestivetract.

Dra. Mª Ángeles VazWinner of the GETHI-Ramón de las Peñas 2013 grant

“Without GETHI’s help, a study like this would have been

very difficult to carry out.”

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CommunicationSharingourchallenges,hopesandachievementsviathedifferentcommunicationchannelshasopenedupnewdoorsforustoaworldofpossibilities,aworldinwhich,thankstothecollaborationofallthosewhobelieveinusandsupportourwork,wearegrowingdaybydayandgaininggroundonraretumours.

WEBSITE

On the GETHI website (www.gethi.org) we provide information about theGroupand its training, socialandresearchactivities,aswellasresourcesandusefultoolsforpatientsorprofessionals,suchastreatmentguidelinesandapps.

Intheprivateareaofthewebsite,GETHImemberscanalsoaccesstheregistries,inwhichtheycanshareandconsultinformationaboutcasesofraretumours

SOCIALNETWORKS

GETHIiswellawareoftheimportanceofsocialnetworksfordisseminationanddiscussionforumsandthereforehasaTwitteraccount:@GrupoGethi.Todate,theGrouphaspublished3,800tweetsandhasmorethan4,900followers.

GETHI IN THE MEDIA

www

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