What is the role of arsenic trioxide in the treatment of acute promyelocytic leukemia (APL)?

Updated: May 26, 2020
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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After studies from China demonstrated that arsenic trioxide was highly active against APL cells in vitro, [92] clinical trials demonstrated that arsenic trioxide resulted in high response rates in patients with relapsed disease. [93, 94] Arsenic trioxide was then moved into the frontline setting, first in combination with standard chemotherapy and then without chemotherapy.

A North American Intergroup study compared the addition of two cycles of consolidation therapy with arsenic trioxide followed by two cycles of chemotherapy with cytarabine and daunorubicin to consolidation with two cycles of cytarabine and daunorubicin chemotherapy without arsenic trioxide. [95] Event-free survival, the primary endpoint, was 77% at 3 years in the arsenic trioxide arm (median not reached) compared with 59% at 3 years in the standard arm (median, 63 mo).

Overall, 84% of adults were alive at last follow-up. Overall survival was 86% at 3 years in the arsenic trioxide arm compared with 77% at 3 years in the standard arm (medians not reached). Maintenance therapy with ATRA, 6-mercaptopurine (6-MP), and methotrexate was effective in preventing relapses compared with no maintenance therapy; however, the optimal schedule of this therapy is not yet determined.

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