Hematopoietic stem cell transplantation from alternative donors for patients with Fanconi anemia and Diamond-Blackfan anemia: a single center experience

Cellular Therapy and Transplantation

Sergey N. Bondarenko¹  Elena V. Babenko¹  Alexander L. Alyanskiy¹  Alexander D. Kulagin¹  Natalia V. Stancheva¹  Elena V. Semenova¹  Ludmila S. Zubarovskaya¹  Irina K. Golubovskaya¹  Boris V. Afanasyev¹ 

关键词: Fanconi anemia;    Diamond-Blackfan anemia;    allogeneic hematopoietic stem cell transplantation;    alternative donor;   

DOI  :  10.3205/ctt-2011-No12-abstract30

学科分类：肿瘤学

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

【 摘 要 】

Aim:Toanalyzetheoutcomesofallogeneichematopoieticstemcelltransplantation(allo-HSCT)fromalternativedonorsonFanconianemia(FA)andDiamond-Blackfananemia(DBA).Methods:BetweenFebruary2007andJune2011sixpatientswithFAandthreepatientswithDBAunderwentalternativedonorallo-HSCT.Themedianpatientagewas9.5years(range3.7–18.5).Eightpatientsreceivedallo-HSCTfrommatchedunrelateddonors.Haploidenticaldonorallo-HSCTwasperformedinonepatientwithFA-associatedacutemyeloidleukemia.Anon-myeloablativefludarabine-containingconditioningregimenwasusedforallpatients.Allpatientsweretransfusion-dependentatthetimeofHSCT.Results:Completedonorengraftmentwasachievedin6patients.OnepatientwithDBAhadinitialautologoushematopoieticrecovery,whichwasfollowedbyasecondtransplantthatresultedin50%donorchimerism.AcuteandchronicGVHDoccurredin5and2patientsrespectively.Sixpatientsarealiveandwellwithamedianfollow-upof11(range1–29)monthsaftertransplant.Thecausesofdeathof3patientswerehemorrhage,acuteGVHDgradeIV(gut,liver),andtransplant-relatedtoxicity.Conclusions:Oursmallstudyconfirmsencouragingresultsofalternativedonorallo-HSCTforpatientswithFAandsteroid-resistanttransfusion-dependentDBA.

【 授权许可】

Unknown