Over the past 2 decades, we have witnessed remarkable progress in the treatment of patients with acute promyelocytic leukemia (APL). The introduction of all-trans retinoic acid (ATRA) in the front-line therapy setting and arsenic trioxide in the relapse setting had already led to a significant improvement in the outcome of these patients. In this randomized trial,1 Dr. Lo-Coco and colleagues have confirmed prior phase II reports of high efficacy of the combination of these two drugs for the initial treatment of patients with low-risk disease, which constitute about two-thirds of the patients with APL. They have unequivocally proven that an acute leukemia can be more effectively treated with drugs that target the underlying pathogenic molecular events rather than with nonspecific cytotoxic chemotherapy, which is also associated with higher toxicity. Although their population was limited to patients with low-risk disease, conventional wisdom as well as prior nonrandomized clinical trials suggest that this strategy, perhaps with the addition of minimal chemotherapy, may also be effective in patients with high-risk APL. More importantly, as the author quite aptly suggests, these results are an important step toward the utilization of targeted therapies for other types of leukemia and hopefully a prelude for future strategies in cancer therapy, in general. ■

Disclosure: Dr. Ravandi reported no potential conflicts of interest.

Reference

1. Lo-Coco F, Avvisati G, Orlando SM, et al: ATRA and arsenic trioxide (ATO) versus ATRA and idarubicin (AIDA) for newly diagnosed, non-high risk acute promyelocytic leukemia (APL): Results of the phase III, prospective, randomized, intergroup APL0406 study led by the Italian-German cooperative groups GIMEMA-SAL_AMLSG. 2012 ASH Annual Meeting. Abstract 6. Presented December 9, 2012.