Established practice in the treatment of patients with acute promyleocytic leukemia and the introduction of arsenic trioxide as a novel therapy.

First-line therapy for patients with newly diagnosed acute promyelocytic leukemia (APL) has been established in a series of randomized clinical trials. Remission induction and consolidation are based on the differentiation agent, all-trans retinoic acid (ATRA), and anthracycline-based chemotherapy. Maintenance therapy is also based on ATRA and may involve additional chemotherapy. Established protocols are associated with a high rate of complete responses (CRs) (87% to 97%), and long-term follow-up has indicated a 4-year disease-free survival of greater than 60%. Therapy for patients who relapse or are refractory to ATRA-based regimens is not standardized and there is a need for new approaches. Arsenic trioxide (ATO) has recently been licensed for use in patients with relapsed/refractory APL. Controlled clinical trials have indicated that ATO is associated with a CR rate of 87% in this population. This agent has a manageable toxicity profile and presents a welcome option for patients with relapsed disease for whom other, more debilitating therapies are unsuitable. Several prognostic factors have been defined in patients with APL, and it is possible that novel treatments such as ATO should be differentially applied to specific prognostic groups. Copyright 2002, Elsevier Science (USA). All rights reserved.