Arsenic trioxide

Background: The ancient drug, arsenic, has remarkable efficacy in the treatment of relapsed acute promyelocytic leukemia (APL) and this success has led to exploration of its use in other malignancies. Objective: To provide an overview of the mechanism of action of arsenic and summarize its development in the treatment of APL and other malignant disorders. Methods: A 20-year search of MEDLINE, EMBASE and Web of Science was conducted. Results/conclusions: A series of clinical trials with arsenic trioxide has confirmed its benefit in the therapy of APL. Its role in the treatment of other malignancies remains to be determined. Careful attention to the clinical management of patients on arsenic trioxide therapy can significantly lessen the risk of major side effects.