11 patients (aged 23–75 years) with refractory acute leukemia were treated at the Maine Medical Center with 5-Azacytidine, 150 mg/m2/day, by continuous infusion for 5 days every 2 weeks. Prior therapy included anthracycline/cytosine arabinoside protocols. Of the 8 patients with refractory de novo acute myelogenous leukemia, 6 achieved remission at an overall response rate of 75% (3 complete remission and 3 partial remission). An average of 1.67 courses was necessary to achieve a response. Remissions were not seen in blastic chronic myelogenous leukemia nor in acute leukemia secondary to cytotoxic drugs. Toxicity included myelo-suppression, moderate nausea and vomiting, abnormal liver function tests, and neuromuscular symptoms. 5-Azacytidine by continuous infusion has significant activity in refractory acute myelogenous leukemia and should be considered for inclusion in primary remission induction therapy.

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