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. 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. 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 = 0.032). 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.
Chen et al. (Fri,) studied this question.