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

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...

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Main Authors: Xianfeng Ouyang, Jianguo Yan, Su Hu, Wenfeng Zhu, Qibao Zhou, Fei Hu
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-05-01
Series:Frontiers in Oncology
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Online Access:https://www.frontiersin.org/articles/10.3389/fonc.2025.1529640/full
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author Xianfeng Ouyang
Jianguo Yan
Su Hu
Wenfeng Zhu
Qibao Zhou
Fei Hu
author_facet Xianfeng Ouyang
Jianguo Yan
Su Hu
Wenfeng Zhu
Qibao Zhou
Fei Hu
author_sort Xianfeng Ouyang
collection DOAJ
description 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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publisher Frontiers Media S.A.
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series Frontiers in Oncology
spelling doaj-art-57d92c8242f6423eb9cb4dff530912c72025-08-20T02:14:15ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2025-05-011510.3389/fonc.2025.15296401529640Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case reportXianfeng Ouyang0Jianguo Yan1Su Hu2Wenfeng Zhu3Qibao Zhou4Fei Hu5Department of Hematology, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaDepartment of Hematology, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaDepartment of Hematology, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaDepartment of Hematology, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaDepartment of Cardiovascular Medicine, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaDepartment of Hematology, Jiujiang First People’s Hospital, Jiujiang, Jiangxi, ChinaMost 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.https://www.frontiersin.org/articles/10.3389/fonc.2025.1529640/fullacute promyelocytic leukemiavariant APLTFG::RARAvenetoclaxall-trans retinoic acidarsenic trioxide
spellingShingle Xianfeng Ouyang
Jianguo Yan
Su Hu
Wenfeng Zhu
Qibao Zhou
Fei Hu
Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
Frontiers in Oncology
acute promyelocytic leukemia
variant APL
TFG::RARA
venetoclax
all-trans retinoic acid
arsenic trioxide
title Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
title_full Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
title_fullStr Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
title_full_unstemmed Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
title_short Venetoclax combined with ATRA shows promising therapeutic potential for TFG:: RARA variant APL: a case report
title_sort venetoclax combined with atra shows promising therapeutic potential for tfg rara variant apl a case report
topic acute promyelocytic leukemia
variant APL
TFG::RARA
venetoclax
all-trans retinoic acid
arsenic trioxide
url https://www.frontiersin.org/articles/10.3389/fonc.2025.1529640/full
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