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Beyond hypomethylating agents failure in patients with myelodysplastic syndromes.


ABSTRACT:

Purpose of review

Although hypomethylating agents (HMAs) significantly improve outcomes in myelodysplastic syndromes (MDS), only half the patients achieve objective responses, and most responders lose response within 1-2 years. Azacitidine prolongs survival by a median of only 9.5 months. Failure of HMA therapy is associated with a very dismal prognosis. Therefore, novel therapeutic approaches are clearly needed.

Recent findings

The sequential use of the alternative HMA after failure of first line HMA is associated with modest efficacy. The improved understanding of the biologic underpinnings of the disease have opened the door to study investigational agents that target disrupted molecular pathways critical to the pathogenesis of MDS. Combination treatment strategies using

SUBMITTER: Zeidan AM 

PROVIDER: S-EPMC4124617 | biostudies-literature | 2014 Mar

REPOSITORIES: biostudies-literature