Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia

Jiong Hu, Yuan Fang Liu, Chuan Feng Wu, Fang Xu, Zhi Xiang Shen, Yong Mei Zhu, Jun Min Li, Wei Tang, Wei Li Zhao, Wen Wu, Hui Ping Sun, Qiu Sheng Chen, Bing Chen, Guang Biao Zhou, Arthur Zelent, Samuel Waxman, Zhen Yi Wang, Sai Juan Chen, Zhu Chen

Research output: Contribution to journalArticle

308 Scopus citations

Abstract

All-trans retinoic acid (ATRA)/arsenic trioxide (ATO) combination-based therapy has benefitted newly diagnosed acute promyelocytic leukemia (APL) in short-term studies, but the long-term efficacy and safety remained unclear. From April 2001, we have followed 85 patients administrated ATRA/ATO with a median follow-up of 70 months. Eighty patients (94.1%) entered complete remission (CR). Kaplan-Meier estimates of the 5-year event-free survival (EFS) and overall survival (OS) for all patients were 89.2% ± 3.4% and 91.7% ± 3.0%, respectively, and the 5-year relapse-free survival (RFS) and OS for patients who achieved CR (n = 80) were 94.8% ± 2.5% and 97.4% ± 1.8%, respectively. Upon ATRA/ATO, prognosis was not influenced by initial white blood cell count, distinct PML-RARα types, or FLT3 mutations. The toxicity profile was mild and reversible. No secondary carcinoma was observed, and 24 months after the last dose of ATRA/ATO, patients had urine arsenic concentrations well below the safety limit. These results demonstrate the high efficacy and minimal toxicity of ATRA/ATO treatment for newly diagnosed APL in long-term follow-up, suggesting a potential frontline therapy for de novo APL.

Original languageEnglish (US)
Pages (from-to)3342-3347
Number of pages6
JournalProceedings of the National Academy of Sciences of the United States of America
Volume106
Issue number9
DOIs
StatePublished - Mar 3 2009

Keywords

  • 5-year EFS
  • 5-year OS
  • Arsenic retention
  • Combination therapy
  • Residual disease

ASJC Scopus subject areas

  • General

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  • Cite this

    Hu, J., Liu, Y. F., Wu, C. F., Xu, F., Shen, Z. X., Zhu, Y. M., Li, J. M., Tang, W., Zhao, W. L., Wu, W., Sun, H. P., Chen, Q. S., Chen, B., Zhou, G. B., Zelent, A., Waxman, S., Wang, Z. Y., Chen, S. J., & Chen, Z. (2009). Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia. Proceedings of the National Academy of Sciences of the United States of America, 106(9), 3342-3347. https://doi.org/10.1073/pnas.0813280106