bone marrow transplant for mds patients .bone marrow transplant for mds patients lori muffly md ms
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BoneMarrowTransplantforMDSPatients
LoriMufflyMDMSAssistantProfessorofMedicine
DivisionofBloodandMarrowTransplantationStanfordUniversity
BMT:Thebasics
BMTforMDS:Theevidence
InnovativeapproachestoBMTforMDS
WhatisBoneMarrow?
WhatisanAllogeneicBMT? BMT=bonemarrow(orstemcell)transplant
Allogeneic=fromaDonor Someoneimmunologicallycompatible
Transplant=Replacerecipient(patients)bonemarrowwiththedonorsbonemarrow ReplaceRBC,platelets,WBC
HowDoWePerformAllogeneicBMT?
Medicaltransplant=NOSURGERY
Howdowecollectdonorstemcells? BonemarrowharvestinOR Usestemcellboosterandcollectviaperipheralveins
STEP1:Conditioning/Prep:PreparesBodyto
AcceptDonorStemCells
STEP2:Infusionof
DonorStemCells
STEP3:StemCellsEngraft
STEP4:Preventsideeffects&re-evaluatemarrowtoensureNOMDS
7-14daysAnhour14-21daysManymonths
WhyDoWePerformAllogeneicBMT?
Transplant=Replacerecipient(patients)bonemarrowwiththedonorsbonemarrow ReplaceRBC,platelets,WBC
New(donor)immunesystemcanbeverypowerfulatcontrollingbloodcancercellsCUREMDS
NewImmuneSystem
NumberofallogeneicBMTs/yr inUShasdoubledinrecentyears
TrendsinAllogeneicBMTUtilizationforAdults70Years,byDisease
AbsoluteNo.HCTs
70Years
0
20
40
60
80
100
120
140
160
AML MDS/MPS Non-Hodgkinlymphoma Others
MufflyetalBlood2017
Historically,patients65andolderwithMedicaredidnothavecoverageforBMTforMDS.
OnAugust4th 2010,theCentersforMedicareandMedicaidservices(CMS)establishedcoverageforBMTforMDSthroughcoveragewithevidencedevelopment(CED).
ACenterforInternationalBoneMarrowTransplantResearch(CIBMTR)studycomparingoutcomesofpatients55-64vs.65andolderwasapprovedinDecember2010.
WhyisAllogeneicBMTforMDSontheRise?
Atallah etalBlood2017
Thestudycomparedtheoutcomesof: 688patientsaged65andolderand 592patientsaged55-64 whounderwentallogeneicBMTforMDSfrom2010-2014
Survivalat100daysandattwoyearsfollowingBMTforMDSpatientsaged65andolderiscomparabletopatientsaged55to64.
AgealoneshouldnotbeadeterminantwhenconsideringBMTforpatientswithMDS.
MedicareCoveragewithEvidenceDevelopmentMDSBMTStudy
Atallah etalBlood2017
HowDoWeDetermineWhichMDSPatienttoTransplant?
DeWitteetalBlood2017
HowDoWeDetermineWhichMDSPatienttoTransplant?
DeWitteetalBlood2017
WhatAreOutcomesafterBMTforMDS?
Ingeneral.
30-40%ofintermediate/highriskMDSpatientswillbecuredfollowingallogeneicBMT
BUT Somepatientswillhaveseriousmorbidity/mortalityfromthetransplantprocessandsomepatientswillhaverecurrenceofprogressionofMDSafterBMT
HowCanWeImproveAllogeneicBMTforMDS?
STEP1:Conditioning/Prep:PreparesBodyto
AcceptDonorStemCells
STEP2:Infusionof
DonorStemCells
STEP3:StemCellsEngraft
STEP4:Preventsideeffects&re-evaluatemarrowtoensureNOMDS
AlterthepreptotargetMDScells
Engineeroroptimizedonor
stemcells
1)ReducetoxicityofallogeneicBMT2)AdditionalMDStargetingpost-
BMT
PhaseI/IIClinicalTrialofanMDSStemCellTargetingAntibodyPlusLowIntensityConditioningfor
PatientswithMDSundergoingAllogeneicBMT
ReducingBMTComplications
PhaseIIIclinicaltrialconductedacrosstheUSaimingtoimprovepost-BMToutcomesforpatientswithMDSandacuteleukemiabyreducingtransplanttoxicity.
Conclusions
AllogeneicBMTisanimmunotherapythatoffersapotentialforcureforintermediate/highriskMDSpatients
TheuseofallogeneicBMTforMDS(andforolderadults)isrising
NewandinnovativeapproachestoBMTareneededtofurtherimproveoutcomes