Arsenic trioxide: Its role in acute promyelocytic leukemia and potential in other hematologic malignancies

The treatment of APL has evolved rapidly. This rare subtype now appears to be highly curable with contemporary strategies which include ATRA plus anthracycline-based chemotherapy. The relapse rate now ranges from approximately 10-30%. Arsenic trioxide has emerged as the treatment of choice for relapsed patients, including those resistant to ATRA. The role of ATO in newly dignosed patients remains under investigation. Its multiple mechanisms of action suggest a role in a wide variety of hematologic malignancies and in vitro and preclinical data support clinical investigators. There is ample in vitro evidence that the efficacy in other diseases may be enhanced by exploiting potential synergy with glutathione reducing agents as well as other novel compunds such as STI-571. AS in the case with most effective antineoplastic agents, the most important clinical benefit may come from combination therapy.