Role of Hypomethylating Agents in the Treatment of Bone Marrow Failure
Technical Report,30 Sep 2014,29 Sep 2015
The Brigham and Womens Hospital, Inc Boston United States
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Hypomethylating agents, 5-azacytidine and decitabine, are effective therapies for the treatment of myelodysplastic syndromesMDS, but the genetic basis of response to these agents is unknown. We hypothesize that, given the heterogeneous clinical response to hypomethylating agents in MDS patients and the diversity of molecular lesions that drive the clinical phenotype of MDS, specific mutations alter response to therapy. The identification of mutations that predict response to therapy could have an immediate clinical impact, enabling physicians to select the patients most likely to respond, to determine the duration of treatment, and to monitor disease progression. Such therapy has the potential to alter the course of disease for patients with bone marrow failure and clonal somatic mutations that occur in myeloid malignancies.