Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report.

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Tác giả: Fei Hu, Su Hu, Xianfeng Ouyang, Jianguo Yan, Qibao Zhou, Wenfeng Zhu

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: Switzerland : Frontiers in oncology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 748854

Most cases of acute promyelocytic leukemia (APL) are driven by the PML::RARA fusion gene, which is sensitive to differentiation induction therapy comprising of all-trans 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.
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