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Identifying effective therapy for myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) is confounded by the marked molecular and cytogenetic abnormalities of these diseases. To determine whether molecular factors determine response to decitabine therapy, investigators performed tumor-cell exome or gene-panel sequencing in 116 patients (age, >60 years) with newly diagnosed AML, relapsed AML, or transfusion-dependent MDS, and then correlated the results with treatment responses.
Higher response rates were observed in patients with unfavorable-risk versus intermediate- or favorable-risk cytogenetics (67% vs. 34%; P<0.001) and in those with TP53 mutation or deletion versus wild-type TP53 (100% vs. 41%; P<0.001). The onset of response…