Objective To explore the survival and prognostic factors of a long-course venetoclax-based (VEN-based) regimen in patients with de novo acute myeloid leukemia (AML) and provide evidence for the maintenance treatment of AML.

Methods A retrospective study was conducted in patients who received a VEN-based regimen and completed at least four courses of efficacy evaluation at The First Affiliated Hospital of Nanchang University from May 2021 to January 2024. The composite complete response rate (cCR), minimal residual disease (MRD)-negative rate, overall survival (OS) time, relapse-free survival (RFS) time, and adverse events were analyzed.

Results Overall, 30 newly diagnosed patients with AML were enrolled in this study. The median age was 65 (range, 53–78) years, and the median number of treatment cycles was 7 (range, 4–20) years. After one cycle, the CR-and MRD-negative rates were 80.0% and 63.3%, respectively. The cumulative cCR was 96.7%, and MRD negative rate was 80.0%, respectively. The median follow-up time was 21.3 (95% confidence intervals 14.7–27.9) months. The median OS time was 32.3 months and RFS time was not reached. The 2-year OS and RFS rates were 70.6% and 54.8%, respectively. Univariate analysis suggested that ELN2017 risk stratification and relapse status affected RFS and OS (P<0.05). However, the multivariate analysis failed to reveal any relationship between these factors and survival (P>0.05). In terms of safety, hematological adverse events were the most common, followed by infections. Overall, the VEN-based regimen was tolerated for patients with AML.

Conclusions A long-course VEN-based regimen is effective and safe. More than half of patients survive for >2 years, and it can be used as an effective maintenance treatment option for patients with AML.