Allogeneic hematopoietic stem cell transplantation with reduced intensity conditioning in children with inherited diseases: a single centre experience

Cellular Therapy and Transplantation

Boris V. Afanasyev¹  Natalia V. Stancheva¹  Natalia I. Zubarovskaya¹  Anastasia S. Borovcova¹  Elena V. Semenova¹  Ludmila S. Zubarovskaya¹  Yulia G. Vasilieva¹ 

关键词: alloHSCT;    inherited diseases;    reduced intensity conditioning;   

DOI  :  10.3205/ctt-2010-No9-abstract38

学科分类：肿瘤学

来源: Universitaetsklinikum Hamburg - Eppendorf / University Medical Center Hamburg - Eppendorf

【 摘 要 】

Background:Allogeneichematopoieticstemcelltransplantation(alloHSCT)isaneffectiveapproachtotreatmentofsomeinheriteddiseases,buttheefficacyofdifferentconditioningregimens(CR)iscontroversial.Conventionalmyeloablativeconditioning(MAC)isassociatedwithhighlevelsoftreatmentrelatedtoxicity(TRT)andwithsignificanttreatmentrelatedmortality(TRM).Reducedintensityconditioning(RIC)significantlydecreasesthedevelopmentofdifferentcomplicationsandreducesTRM.ButRICisassociatedwithanincreasedfrequencyofgraftfailureorallograftrejectioninthesediseases.Purpose:ToestimatetheefficacyofRICinpatients(pts)withinheriteddiseases.Patients:FromMay2006toMay2010MUDalloHSCTwasperformedin11ptswiththefollowingdiseases:Wiscott-Aldrichsyndrome,2,Krabbedisease,1,metachromaticleukodystrophy(ML),1,X-linkedadrenoleukodystrophy(xALD),1,malignantosteopetrosis,1,Diamond-Blackfananemia(DBA),1,Hurlersyndrome,1,andFanconianemia(FA),3.Medianptagewas5.9years.WeusedFludarabine(Flu)150mg/m2+Melphalan140mg/m2in5pts;Flu150mg/m2+Cyclophosphamide(Cy)600mg/m2in1ptwithDBA;andFlu150mg/m2+Cy40mg/kgin3ptswithFA.aGvHDprophylaxiswasbyCsA+MTX+ATGAM.Results:Three-yearoverallsurvivalis75%.TenptsengraftedonD+14(rangeD+12–19)withfulldonorchimerismachievementonD+30.8ptsarealivewithoutanysignsofdiseasewithfollow-upat1–49months.TheptwithMLdiedonD+37fromprogressiondisease;2pts(1withFA,1withxALD)diedfromseveregutaGvHD.DuringRICnobodydevelopedseveretoxicity(III–IVstaccordingWHOclassification).Conclusion:RICiseffectiveinchildrenwithinheriteddiseases,haslowTRTandisapromisingalternativetoMAC.

【 授权许可】

Unknown