AML Induction Therapy with Outpatient Azacitidine.

15 patients with newly diagnosed acute myelogenous leukemia were treated with outpatient 5-aza-2′-deoxycytidine (azacitidine, 75 mg/m² subcutaneously for seven days every four weeks) as primary induction therapy. The average age was 68 years (range = 44–80). Bone marrow blast counts ranged between 20% and 38%. The overall response rate (complete remission, CR + parital remission, PR) was 53% (8/15). CR was achieved in 4 patients (27%) in an average of 3 cycles (range = 2–5). Duration of response averaged 8 cycles (range = 6–13) from the time CR was documented. Two of these patients went on to allogeneic hematopoietic stem cell transplantation (one in CR, one in PR). PR was achieved in 4 patients (27%) in an average of 3 cycles (range = 2–3). Duration of response averaged 7 cycles (range = 1–15) from the time PR was documented. Two patients continue therapy, maintaining PR, at 9 and 15 cycles. One patient met criteria for stable disease with a survival of 17 months. One patient with biphenotypic leukemia survived 12 months with a significant reduction in transfusion requirements. One patient demonstrated a major platelet response, but discontinued therapy after 3 cycles due to anorexia. Four patients had disease progression after 1, 1, 2, and 3 cycles. The average survival for patients who achieved at least stable disease and did not go on to hematopoietic stem cell transplantation was 14⁺ months (range = 8–24). None of the responders had an ECOG performance status worse than 1 while being treated. The most common toxicity was febrile neutropenia (4 patients, 2 deaths). Azacitidine administered in the outpatient setting can induce remission in AML. This therapy appears to be well tolerated. Further study is warranted in poor risk patients to document activity and toxicity.