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Abstract
Over the last 17 years, clinical trials conducted worldwide have demonstrated the
efficacy of arsenic trioxide (As(2)O(3)) in the treatment of relapsed acute promyelocytic
leukemia (APL). Currently, the role of As(2)O(3) in front-line therapy is under investigation.
Recent trials in the US have demonstrated that the addition of As(2)O(3) to standard
treatment regimens improves survival outcomes in patients with APL and may allow a
reduction in cytotoxic chemotherapy exposure. As(2)O(3) has also shown efficacy in
other malignancies, particularly multiple myeloma and myelodysplastic syndromes. Therapeutic
doses of As(2)O(3) are well tolerated, with no evidence of long-term toxicity. Adverse
events include APL differentiation syndrome, electrocardiographic abnormalities, and
mild elevations in liver enzymes. This review highlights trials investigating the
role of As(2)O(3) in induction and consolidation for newly diagnosed APL, as well
as its role in other hematologic malignancies. The chemistry, mechanisms of action,
and clinical side effects of As(2)O(3) are also discussed.
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