A randomized phase II/III study of 'novel therapeutics' versus azacitidine in newly diagnosed patients with acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CMML), age 60 or older: a report of the comparison of azacitidine and nivolumab to azacitidine: SWOG S1612

Azacitidine (AZA) treatment prolongs overall survival (OS) in higher-risk MDS patients compared with conventional care regimens (CCR): results of the AZA-001 phase III study. Acute myeloid leukemia (AML) primarily affects older adults, for whom lower intensity therapies, such as the hypomethylating agents (HMAs) azacitidine and decitabine, or best supportive care have often been selected. In arm B, three patients had an FLT3-ITD mutation and died within 28 days of therapy initiation, one patient with FLT3-ITD mutation was randomized to the azacitidine alone arm. The study was closed prematurely after an observed excess early mortality in arm B, where six patients (24%) died within 28 days of initiating therapy versus one patient (4%) in arm A ( I p i = 0.098). [Extracted from the article]