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: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2025-05-01
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| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fonc.2025.1529640/full |
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| Summary: | 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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| ISSN: | 2234-943X |