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

Open access
Hu, Jiong, Liu, Yuan-Fang, Wu, Chuan-Feng, Xu, Fang, Shen, Zhi-Xiang, Zhu, Yong-Mei, Li, Jun-Min, Tang, Wei, Zhao, Wei-Li, Wu, Wen, Sun, Hui-Ping, Chen, Qiu-Sheng, Chen, Bing, Zhou, Guang-Biao, Zelent, Arthur, Waxman, Samuel, Wang, Zhen-Yi, Chen, Sai-Juan, Chen, Zhu·2009·Proceedings of the National Academy of Sciences

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-RAR α 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.

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Metadata

Authors
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.
Venue
Proceedings of the National Academy of Sciences
Published
2009-03-03
Volume
106
Issue
9
Pages
3342-3347
ISSN
  • 0027-8424 (ISSN-L)
  • 1091-6490
OpenAlex
W1993816996
Cited by
425
References
0
FWCI
14.16
Type
journal article
Language
en
Open access
green

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CoChat. (2026). Citation check: Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia. https://app.cochat.ai/papers/10.1073/pnas.0813280106
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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. https://doi.org/10.1073/pnas.0813280106

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