Real-life Multicenter Experience of Long-term Treatment With Venetoclax Plus Azacitidine for Acute Myeloid Leukemia in China

Background The combination of venetoclax (VEN) and azacitidine (AZA) has transformed the treatment of acute myeloid leukemia (AML) in patients ineligible for intensive therapy. Nevertheless, real-world evidence on long-term outcomes in patients with AML treated with VEN + AZA remains limited. Methods A retrospective analysis was conducted on 79 patients with naïve AML who received at least 6 cycles of VEN + AZA across five collaborating hematology units in China between September 2021 and October 2024. Results The median age at diagnosis was 67 years (range: 49–87), and the median number of VEN treatment cycles was 9 (range: 6–20). The CR/CRi occurred in 56 of 79 patients (70.9%), with higher rates observed in those harboring NPM1 (80.9%) and IDH2 (80.0%) mutations. The median overall survival (OS) reached 33.5 months, with a median follow-up of 14 months and a median duration of response of 25.4 months. Patients aged over 65 demonstrated inferior OS compared with younger patients (median OS: 26.1 months and not reached, respectively). Patients with NPM1 and IDH2 mutations were associated with improved OS, although the difference did not reach statistical significance. Patients treated with a lower dose of VEN achieved superior OS compared to those receiving the standard dose (P = 0.019). Conclusion Prolonged VEN + AZA therapy is effective for AML, achieving sustained remission in a subset of patients and supporting its role as a viable maintenance therapy in this population.