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Long-course venetoclax with hypomethylating agents in unfit AML patients shows stable efficacy and MRD prognostic value.

Long-course venetoclax with hypomethylating agents in unfit AML patients shows stable efficacy and M…
Photo by Dmytro Vynohradov / Unsplash
Key Takeaway
Consider long-course venetoclax plus hypomethylating agents for unfit AML, noting need for prospective validation.

This single-center retrospective analysis assessed long-course venetoclax combined with hypomethylating agents in unfit AML patients. The sample size was not reported, and follow-up duration was not reported. The study setting was single-center. Study phase was not reported.

The intervention involved long-course venetoclax combined with hypomethylating agents. No comparator was reported. Clinical efficacy was stable, and disease remission was effectively achieved. MRD negativity was closely associated with better prognosis, whereas higher recurrence risk was noted. Secondary outcomes included efficacy, safety, and prognostic value of measurable residual disease. Correlation between dynamic MRD changes and patient prognosis was assessed.

Safety included hematological and infectious non-hematological toxicity with controllable overall safety. Serious adverse events were not reported. Limitations include single-center design, limited sample size, absence of a short-course control group, and survivorship bias should be considered. Survivorship bias should be considered in interpretation.

Practice relevance involves providing evidence for optimizing clinical low-intensity treatment regimens. Causal conclusions about treatment duration cannot be drawn. Conclusions require validation by larger multicenter prospective studies. Generalizability is limited due to single-center design and limited sample size. Funding or conflicts were not reported. Do not overstate causal conclusions about treatment duration. Certainty requires external validation.

Study Details

Study typeCohort
EvidenceLevel 3
PublishedApr 2026
View Original 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.
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