Current first- and second-line treatment options in acute promyelocytic leukemia

Abstract

Outcome of acute promyelocytic leukemia (APL) has remarkably improved during the last 30 years, especially after the identification of PML–RARA oncogene as a key in the pathogenesis of APL and all-trans retinoic acid as therapeutic agent. Arsenic trioxide has been recently demonstrated to be the most effective single antileukemic agent and it has also showed synergistic action when combined with all-trans retinoic acid, decreasing relapse rate especially in low/intermediate-risk settings. Therapeutic advances led to complete remission rates of more than 90%, modifying disease history. In relapse setting, arsenic trioxide-based regimens showed efficacy for the achievement of second molecular complete remission. The most challenging issue in APL management remains the significant early deaths rate, nowadays the principal reason for treatment failure.