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Prolonged treatment-free remission after venetoclax in combination with a hypomethylating agent in acute myeloid leukemia with inv(16)
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DOI：10.1007/s00277-026-07237-0.png)
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Abstract
En 中文
Core binding factor acute myeloid leukemia (CBF-AML)—defined by t(8;21) or inv(16) chromosomal rearrangements—is classified as favorable-risk disease per the European LeukemiaNet 2022 risk model and is typically treated with intensive chemotherapy. Patients unsuitable for intensive therapy increasingly receive hypomethylating agents plus venetoclax (HMA/ven), though CBF-AML was excluded from pivotal trials of this regimen. Furthermore, HMA/ven is typically administered as indefinite therapy in AML; however, there is increasing interest in exploring the feasibility of therapy cessation given persistent treatment-free remissions with this regimen. Here, we report the first case of sustained treatment-free remission following HMA/ven in CBF-AML. Features in this case that suggest a higher likelihood of achieving a durable treatment-free remission include inv(16) disease and rapid measurable residual disease (MRD) clearance. We summarize the current retrospective data comparing response rates of inv(16) vs. t(8;21) AML to HMA/ven: inv(16) AML consistently exhibits higher response rates compared with t(8;21) disease, although an overall survival benefit is not well established. Emerging data also suggest achieving MRD-negative complete remission after induction may predict more durable outcomes. This case provides proof-of-concept that carefully selected CBF-AML patients achieving sustained MRD-negative remission with HMA/ven can successfully discontinue therapy. Prospective evaluation of HMA/ven in unfit CBF-AML patients, weighed against dose-attenuated intensive chemotherapy, is needed to define its role and optimal duration.
Keywords:
Core binding factor acute myeloid leukemia (CBF-AML)
Treatment-free remission
Hypomethylating agent
Venetoclax
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