5-Azacytidine: A New Active Agent for the Treatment of Acute Leukemia

Thirty-seven children with acute leukemia were treated with 5-azacytidine in 5-day courses given every 14 days. Six out of 14 children with acute myelogenous leukemia who were adequately treated achieved an M₁ marrow. Five of these subsequently developed complete remissions lasting 8 mo, 6 mo, 3 mo, 2 mo, and 2 mo. Of 22 children with acute lymphocytic leukemia, one achieved an M₁ marrow and one an M₂ marrow. The former attained a complete remission which lasted 3 mo. The maximum tolerated dose is between 150 to 200 mg/sq m on a daily x 5 schedule given every 14 days. The impressive activity of 5-aza-C in patients with acute myelogenous leukemia resistant to cytosine arabinoside indicates that this drug will become an important addition to the therapeutic armamentaria against this type of leukemia.