bone marrow transplant for mds patients .bone marrow transplant for mds patients lori muffly md ms

Download 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

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