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Maintenance With Oral Azacitidine Extends Survival in Older Patients With AML in First Remission

In a phase 3 study of older patients with acute myeloid leukemia (AML) in remission after chemotherapy, maintenance therapy with CC-486—the oral formulation of azacytidine—was associated with significantly longer overall and relapse-free survival than placebo (N Engl J Med. 2020;383:2526-2537).

“Although induction chemotherapy results in remission in many older patients with acute myeloid leukemia (AML), relapse is common and overall survival is poor,” wrote Andrew H. Wei, MB, BS, PhD, Department of Clinical Haematology, Alfred Hospital, Melbourne, Australia, and colleagues.

Using a cohort of 472 patients (aged ≥55 years) with AML in first remission after intensive chemotherapy, Dr Wei et al conducted a double-blind, placebo-controlled trial of maintenance therapy with CC-486 (a hypomethylating agent not bioequivalent to injectable azacitidine).

These patients (median age, 68 years) were randomized to receive 300 mg of CC-486 (n = 238) or placebo (n = 234) once daily for 14 days per 28-day cycle.

The primary end point of the study was overall survival, and secondary end points included relapse-free survival and health-related quality of life.

From the time of randomization, the median overall survival was significantly longer with CC-486 than with placebo (24.7 months vs 14.8 months, respectively; P <.001). In addition, the median relapse-free survival was also significantly longer with CC-486 than with placebo (10.2 months vs 4.8 months, respectively; P <.001).

“Benefits of CC-486 with respect to overall and relapse-free survival were shown in most subgroups defined according to baseline characteristics,” the investigators reported.

The most frequently reported adverse events in both treatment arms were grade 1 or 2 gastrointestinal events. Neutropenia (41% in the CC-486 group and 24% in the placebo group) and thrombocytopenia (22% and 21%, respectively) were the most common grade 3 or 4 adverse events.

Of note, during therapy with CC-486, the overall health-related quality of life was preserved.

“CC-486 maintenance therapy was associated with significantly longer overall and relapse-free survival than placebo among older patients with AML who were in remission after chemotherapy,” Dr Wei and co-investigators concluded.

“Side effects were mainly gastrointestinal symptoms and neutropenia. Quality-of-life measures were maintained throughout treatment,” they added.—Hina M. Porcelli

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