OBJECTIVETo summarize the clinical applications of arsenic trioxide (ATO) in the treatment of acute promyelocytic leukemia (APL), as well as non-APL malignancies and to discuss the mechanisms and adverse effects involved in ATO administration.

DATA SOURCESThe data in this article were collected from PubMed and CHKD database with relevant English and Chinese articles published from 1957 to 2011, with key words including acute promyelocytic leukemia, arsenic trioxide, treatment, and mechanism.

STUDY SELECTIONArticles including any information about ATO in the treatment of APL were selected.

RESULTSAPL is a rare subtype of acute myeloid leukemia, with dismal prognosis under treatment with traditional chemotherapy. ATO impressively increases the complete remission rate and prolongs survival of patients with APL, with only mild and transient adverse effects. The advances in the understanding of multiple mechanisms involved in ATO treatment will benefit more cancers in future.

CONCLUSIONDeeper understanding of mechanisms involved in ATO treatment may provide rationales for future clinical applications in a number of human malignancies.