This post hoc analysis of the DACO-016 phase III study evaluates the impact of decitabine on transfusion dependence and survival in 485 elderly patients with newly diagnosed acute myeloid leukemia (AML). Red blood cell (RBC) and platelet (PLT) transfusion independence, defined as no transfusions for ≥ 8 consecutive weeks, was measured in both decitabine (n = 242) and treatment choice (TC, n = 243) arms. More RBC transfusion dependent patients at baseline became transfusion independent with decitabine than with TC (26% vs. 13%; p = 0.0026). Similar results were obtained for patients who were PLT transfusion dependent at baseline (31% vs. 13%; p = 0.0069). When excluding patients who attained complete remission (CR), survival was improved in patients who achieved RBC or PLT transfusion independence, suggesting that reaching CR is not a prerequisite for deriving benefit from treatment with decitabine. In addition, patients who achieved transfusion independence with decitabine had increased treatment continuation, even in the absence of CR.
Keywords: Myeloid leukemias and dysplasias; decitabine; survival; transfusion dependence.