Abstract
Front-line treatment with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) is superior to standard ATRA and chemotherapy (CHT) in patients with low-/intermediate-risk acute promyelocytic leukemia (APL). However, for high-risk (HR) patients (defined as those with a white blood cell count ≥ 10×10⁹/L), the role of ATRA-ATO is subject to debate, and study data are scarce. The objective for the present real-world cohort study was to assess the outcomes in 135 HR APL patients treated with ATRA-CHT or ATRA-ATO during induction at 12 French hospitals between 2010 and 2021. Of the 135 patients, 50 (37%) received ATRA-ATO as induction therapy (though combination with cytoreductive treatment was usually necessary), and 85 received standard ATRA-CHT (“CHT group”). The complete response rates were 90% in the ATRA-ATO group and 76% in the CHT group (p = 0.052). The five-year overall survival (OS) rate was significantly higher in the ATRA-ATO group (89.86% [95% confidence interval: 81.81–98.70]) than in the CHT group (72.69% [63.30–83.47]; p = 0.035). The combination of ATRA and ATO was effective and safe in this large, real-world cohort of HR APL patients. The forthcoming results of the APOLLO trial (a direct comparison of ATRA-ATO with ATRA-CHT) might validate our present findings.
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LA and DL designed the study; DL and AD collected, controlled the database and analyzed data; BC, MH, SB, CB, AZ, ML, CB, ND, CW, MPGH, MO, TC, FC, AC, CR, MHB, ER, PF included and treated patients; AD performed the statistical analysis; AD, LA and DL wrote the manuscript. All authors revised the manuscript for critical content and approved the submitted version.
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The study was conducted in accordance with the Declaration of Helsinki. The study protocol was approved by the French National Consultative Committee on Information Processing in Medical Research (Comité Consultatif sur le Traitement de l’Information en matière de Recherche dans le domaine de la Santé, Paris, France; reference: 15.414), and the study database was registered with the French National Data Protection Commission (Commission nationale de l’informatique et des libertés, Paris, France; reference: DR-2015-534). Informed consent was obtained from all participants.
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Lebon, D., Da Rocha, A., Cassinat, B. et al. Comparison of induction with arsenic trioxide or chemotherapy in a real-world cohort of patients with high-risk acute promyelocytic leukemia. Leukemia 39, 1865–1870 (2025). https://doi.org/10.1038/s41375-025-02663-x
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DOI: https://doi.org/10.1038/s41375-025-02663-x
