Treatment of acquired aplastic anemia in children

Cellular Therapy and Transplantation

PhD¹  Seiji Kojima MD¹ 

关键词: aplastic anemia;    children;    ATG;    UBMT;    HLA;   

DOI  :  10.3205/ctt-2010-No9-abstract64

学科分类：肿瘤学

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

【 摘 要 】

Themajortreatmentoptionsforpatientswithsevereaplasticanemia(AA)includeallogeneicstemcelltransplantation(SCT)andimmunosuppressivetherapy(IST).BecausetheresponseratetoISTrangesfrom60to70%,30to40%ofpatientsrequiresecondlinetherapiessuchasasecondroundofISTorSCTfromalternativedonors.WeprospectivelycomparedtheefficacyofrepeatedISTwithSCTfromanalternativedonorinnon-responderstoaninitialcourseofIST.Failurefreesurvival(FFS)wasmuchbetterintheSCTgroupcomparedwiththeISTgroup,whichsuggeststhatSCTfromanalternativedonoroffersabetterchanceofFFSthanasecondIST.ToimproveoutcomesinAApatientswhoreceiveunrelatedbonemarrowtransplantation(UBMT),itiscrucialtodefineacceptableHLAmismatchingoftheunrelateddonor.WeretrospectivelyanalyzedtheimpactofHLAmismatching(HLA-A,-B,-C,-DRB1,-DQB1)onthetransplantoutcomefor301patientswithAAwhoreceivedUBMTthroughtheJapanMarrowDonorProgram.TheadditionalimpactofHLA-DPB1wasanalyzedfor10/10or9/10matchedpairs(n=169).Theprobabilityofoverallsurvival(OS)at5yearswascomparableamongcompletematchedpairsandHLA1allelemismatchedpairs.Incontrast,OSintransplantsfromadonorwith2ormoreallelemismatcheswassignificantlyworse.HLA-DPB-1mismatchingdidnotprovideanyadditionaleffectonsurvival,acute,andchronicGVHD.A10/10matchedunrelateddonorisoptimal,but9/10(anylocus)matchedunrelateddonorsareacceptableforpatientswithAA.Optimizationofdonorselection,conditioningregimen,andGVHDprophylaxiswillimprovetheoutcomeofAApatientswhoreceivebonemarrowtransplantationfromanunrelateddonor.

【 授权许可】

Unknown