Intravenous arsenic trioxide and all-trans retinoic acid as front-line therapy for low-risk acute promyelocytic leukemia

Introduction: The outcome of acute promyelocytic leukemia (APL) has drastically improved following the identification of the PML-RARA oncogene as a key player in the pathogenesis of APL, and the subsequent introduction of all-trans retinoic acid (ATRA) as a therapeutic agent. Areas covered: Randomized trials have recently demonstrated the efficacy of arsenic trioxide (ATO) in combination with ATRA for the front-line treatment of standard and medium risk APL patients. This chemotherapy-free combination is associated with a decreased cumulative rate of relapse, prolonged overall survival, and reduced early death rate. Expert commentary: The most challenging issue in the management of APL remains the significant rate of early deaths in high-risk patients. The ongoing studies will clarify the possible role of ATO in this setting in combination with ATRA and other agents. The aim of this review is to report data of efficacy and safety of intravenous ATO in newly diagnosed patients and discuss on its potential role as a new standard of care for APL patients.