A real-world data study on long-course venetoclax combined with hypomethylating agents in unfit patients

This study aimed to investigate the efficacy, safety and prognostic value of measurable residual disease (MRD) in unfit AML patients treated with long-course venetoclax combined with hypomethylating agents, so as to provide evidence for optimizing clinical low-intensity treatment regimens.A single-center retrospective analysis was conducted on unfit AML patients receiving this regimen. Clinical data were collected to explore the correlation between dynamic MRD changes at different treatment stages and patient prognosis.The long-course regimen exhibited stable clinical efficacy and effectively achieved disease remission. MRD negativity was closely associated with better prognosis, while patients with MDS-transformed AML and relapsed/refractory AML had higher recurrence risk. Adverse reactions were mainly hematological and infectious non-hematological toxicity, with controllable overall safety.Dynamic MRD monitoring provides valuable prognostic information in unfit AML patients receiving venetoclax plus hypomethylating agents. Sustained MRD negativity predicted favorable outcomes, highlighting the clinical utility of MRD-guided strategies. Due to the absence of a short-course control group, causal conclusions about treatment duration cannot be drawn, and survivorship bias should be considered. Study limitations include single-center design and limited sample size; conclusions require validation by larger multicenter prospective studies.

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Publication Details

Journal
Frontiers in Oncology
Published
2026-04-21
DOI
https://doi.org/10.3389/fonc.2026.1826350
Primary Topic
Acute Myeloid Leukemia Research
Type
article
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article

A real-world data study on long-course venetoclax combined with hypomethylating agents in unfit patients

Minran Zhou, X Li, Xinwen Jiang, Q S Ji et al.
Frontiers in Oncology
Acute Myeloid Leukemia Research
article

A real-world data study on long-course venetoclax combined with hypomethylating agents in unfit patients

Minran Zhou, X Li, Xinwen Jiang, Q S Ji, Chen Cao, Sai Ma, Chunyan Chen
article en

Abstract

This study aimed to investigate the efficacy, safety and prognostic value of measurable residual disease (MRD) in unfit AML patients treated with long-course venetoclax combined with hypomethylating agents, so as to provide evidence for optimizing clinical low-intensity treatment regimens.A single-center retrospective analysis was conducted on unfit AML patients receiving this regimen. Clinical data were collected to explore the correlation between dynamic MRD changes at different treatment stages and patient prognosis.The long-course regimen exhibited stable clinical efficacy and effectively achieved disease remission. MRD negativity was closely associated with better prognosis, while patients with MDS-transformed AML and relapsed/refractory AML had higher recurrence risk. Adverse reactions were mainly hematological and infectious non-hematological toxicity, with controllable overall safety.Dynamic MRD monitoring provides valuable prognostic information in unfit AML patients receiving venetoclax plus hypomethylating agents. Sustained MRD negativity predicted favorable outcomes, highlighting the clinical utility of MRD-guided strategies. Due to the absence of a short-course control group, causal conclusions about treatment duration cannot be drawn, and survivorship bias should be considered. Study limitations include single-center design and limited sample size; conclusions require validation by larger multicenter prospective studies.

Frontiers in OncologyVol. 16
Microelectronica (Romania) (RO), Shandong Provincial Hospital (CN), Qilu Hospital of Shandong University (CN)
Good health and well-being
Openalex Percentile: Top 19%
Acute Myeloid Leukemia Research
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