International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts.

This multicenter, randomized, open-label, phase 3 trial evaluated azacitidine efficacy and safety vs conventional care regimens (CCR) in 488 patients aged ≥65 years with newly diagnosed acute myeloid leukemia (AML) with >30% bone marrow blasts. Before randomization, a CCR (standard induction chemotherapy, low-dose cytarabine, or supportive care only) was preselected for each patient. Patients then were assigned 1:1 to azacitidine (n=241) or CCR (n=247). Patients assigned to CCR received their preselected treatment. Median overall survival (OS) was increased with azacitidine vs CCR: 10.4 (95%CI: 8.0-12.7) vs 6.5 (95%CI: 5.0-8.6) months, respectively; hazard ratio (HR) 0.85 (95%CI: 0.69-1.03), stratified log-rank P = .1009. One-year survival rates with azacitidine and CCR were 46.5% and 34.2%, respectively (difference 12.3% [95%CI: 3.5%-21.0%]). A prespecified analysis censoring patients who received AML treatment after discontinuing study drug showed median OS with azacitidine vs CCR was 12.1 (95%CI: 9.2-14.2) vs 6.9 (95%CI: 5.1-9.6) months; HR 0.76 (95%CI: 0.60-0.96), stratified log-rank P = .0190. Univariate analysis showed favorable trends for azacitidine compared with CCR across all subgroups defined by baseline demographic and disease features. Adverse events were consistent with the well-established safety profile of azacitidine. Azacitidine may be an important treatment option for this difficult-to-treat AML population. [ABSTRACT FROM AUTHOR]