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CASE REPORT article
Front. Oncol.
Sec. Hematologic Malignancies
Volume 15 - 2025 | doi: 10.3389/fonc.2025.1529640
This article is part of the Research TopicCombination Therapies in Acute Myeloid Leukemia (AML).View all 5 articles
Venetoclax combined with ATRA shows promising therapeutic potential for TFG::RARA variant APL：a case report
Provisionally accepted- Jiujiang First People's Hospital, Jiujiang, Jiangxi Province, China
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Most cases of acute promyelocytic leukemia (APL) are driven by the PML::RARA fusion gene, which is sensitive to differentiation induction therapy comprising of alltrans retinoic acid (ATRA) and arsenic trioxide (ATO). Treatment with ATRA plus ATO has achieved remarkable clinical outcome in patients with typical APL. However, 5% of patients still died from relapsed/refractory disease, predominantly high-risk APL and variant APL. The diagnosis and treatment of variant APL remain challenging. Here, we report a case of TFG::RARA variant APL recognized by targeted RNA sequencing. The patient achieved a sustained complete response following treatment with venetoclax combined with ATRA. Currently, the overall survival (OS) of the patient has exceeded 30 months, and the progression-free survival (PFS) has reached 29 months. Our results suggest that venetoclax combined with ATRA may be an ideal treatment option for patients with TFG::RARA variant APL.
Keywords: Acute promyelocytic leukemia, Variant APL, TFG::RARA, venetoclax, All-trans retinoic acid, arsenic trioxide
Received: 17 Nov 2024; Accepted: 09 Apr 2025.
Copyright: © 2025 Ouyang, Yan, Hu, Zhu, Zhou and Hu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Fei Hu, Jiujiang First People's Hospital, Jiujiang, Jiangxi Province, China
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