This multicenter, randomized, open-label, phase 3 trial evaluated azacitidine efficacy and safety vs conventional care regimens (CCRs) in 488 patients age ≥65 years with newly diagnosed acute myeloid leukemia (AML) with >30% bone marrow blasts. Before randomization, a CCR (standard induction chemotherapy, low-dose ara-c, 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 months (95% confidence interval [CI], 8.0-12.7 months) vs 6.5 months (95% CI, 5.0-8.6 months), respectively (hazard ratio [HR] was 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 months (95% CI, 9.2-14.2 months) vs 6.9 months (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. This trial was registered at www.clinicaltrials.gov as #NCT01074047.
International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts / Dombret, H.; Seymour, J. F.; Butrym, A.; Wierzbowska, A.; Selleslag, D.; Jang, J. H.; Kumar, R.; Cavenagh, J.; Schuh, A. C.; Candoni, A.; Recher, C.; Sandhu, I.; Del Castillo, T. B.; Al-Ali, H. K.; Martinelli, G.; Falantes, J.; Noppeney, R.; Stone, R. M.; Minden, M. D.; Mcintyre, H.; Songer, S.; Lucy, L. M.; Beach, C. L.; Dohner, H.. - In: BLOOD. - ISSN 0006-4971. - 126:3(2015), pp. 291-299. [10.1182/blood-2015-01-621664]