An Erythroid Differentiation Signature Predicts Response to Lenalidomide in Myelodysplastic Syndrome 英文参考文献.docVIP
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An Erythroid Differentiation Signature Predicts Response to Lenalidomide in Myelodysplastic Syndrome 英文参考文献
o
PL SMEDICINE
AnErythroidDifferentiationSignaturePredicts
ResponsetoLenalidomideinMyelodysplastic
Syndrome
Benjamin L.Ebert1,2,3[,Naomi Galili4[,Pablo Tamayo1,Jocelyn Bosco1,2,Raymond Mak1,2,Jennifer Pretz1,2
Shyam Tanguturi1,Christine Ladd-Acosta1,Richard Stone2,3,Todd R.Golub1,2,5,6,Azra Raza4*
,
1 Broad Institute of Harvard and Massachusetts Institute of Technology, Cambridge, Massachusetts, United States of America, 2 Dana-Farber Cancer Institute, Harvard
Medical School, Boston, Massachusetts, United States of America, 3 Brigham and Women’s Hospital, Department of Medicine, Boston, Massachusetts, United States of
America, 4 St. Vincent’s Comprehensive Cancer Center, New York, New York, United States of America, 5 Childrens’s Hospital, Boston, Massachusetts, United States of
America,6HowardHughesMedicalInstitute,ChevyChase,Maryland,UnitedStatesofAmerica
Funding:Thisworkwassupported
bytheHowardHughesMedical
Instituteandgrant1R01-HG003945–
01toTRG,theRadheyKhannaMDS
researchgrant,andtheShyamalan
FoundationgranttoAR,andK08-
HL078818–01toBLE.Thefunders
hadnoroleinstudydesign,data
collection/analysis,decisionto
publish,orpreparationofthe
manuscript.
ABSTRACT
Background
Lenalidomideisaneffectivenewagentforthetreatmentofpatientswithmyelodysplastic
syndrome (MDS), an acquired hematopoietic disorder characterized by ineffective blood cell
productionandapredispositiontothedevelopmentofleukemia.Patientswithaninterstitial
deletion of Chromosome 5q have a high rate of response to lenalidomide, but most MDS
patients lack this deletion. Approximately 25% of patients without 5q deletions also benefit
from lenalidomide therapy, but response in these patients cannot be predicted by any
currentlyavailablediagnosticassays.Theaimofthisstudywastodevelopamethodtopredict
lenalidomide response in order to avoid unnecessary toxicity in patients unlikely to benefit
fromtreatment.
CompetingInterests:AzraRazaand
RichardStoneareinvolvedwitha
speaker’sbureauforCelgene.
AcademicEditor:F
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