Azacitidine in patients with acute myeloid leukemia medically unfit for or resistant to chemotherapy: a multicenter phase I/II study

Abstract

The safety and efficacy of azacitidine (5-day schedule) were assessed in a multicenter study in 40 patients (median age 72 years) with acute myeloid leukemia (AML) medically unfit for (n = 20) or resistant to chemotherapy (n = 20) from April to October 2008. Median marrow blasts were 42%. After a median follow-up of 13 months, response (complete remission [CR]/partial remission [PR]/hematologic improvement [HI]) was 50% and 10% in newly diagnosed and relapsed/refractory patients, respectively (p = 0.008). Median time-to-response was 2.5 months with a median duration of 5.9 months. Median survival was not reached for responders versus 3.8 months for 15 (38%) patients with stable disease (p < 0.045). High-risk cytogenetics was associated with inferior survival (p = 0.05). Lower marrow blasts on day 15 of cycle 1, irrespective of pretreatment count, predicted subsequent response (p = 0.01). Azacitidine is active and well tolerated in elderly patients with newly diagnosed AML.

Keywords::

• Acute myeloid leukemia
• azacitidine treatment
• elderly patients
• hypomethylating agents

Acknowledgements

The authors received editorial support from Excerpta Medica in the preparation of this manuscript, funded by Celgene Corporation. The authors are fully responsible for content and editorial decisions for this manuscript.

H.K.A-A. has received honoraria from Celgene Corporation, and research funding from Celgene Corporation and Chugai Pharma.

Potential conflict of interest:

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