Introduction: Acute promyelocytic leukemia (APL), the most rapidly fatal leukemia only two decades ago, has been converted into the most frequently curable leukemia by the advent of all-trans retinoic acid (ATRA) and its combination with anthracycline-based chemotherapy. More recently, arsenic trioxide (ATO) has been shown to be the most effective single agent in this disease and has been approved for the treatment of relapsed patients both in the United States and Europe. Moreover, ATO has been included in the design of several front-line studies, with the aim to reduce therapy-related toxicity while maintaining the potential of cure.
Areas covered: First, this review briefly discusses the mechanisms of action and the toxicity profile of ATO. Furthermore, the reported experience on the use of ATO as single agent or in combinatorial schemes both in relapsed and in newly diagnosed patients with APL is critically reviewed. Finally, the use of this agent in special subsets of patients unfit to receive conventional chemotherapy is discussed, along with its potential role in maintenance therapy.
Expert opinion: While the role of ATO as single agent or in combination with ATRA is well established and recommended by the European LeukemiaNet guidelines as a first option for relapsed patients, the role of the drug in newly diagnosed patients is still uncertain and based only on evidence levels mostly originating from non-randomized trials. The results of ongoing randomized studies should better define the role of ATO in front-line therapy.