Real-world survival analysis of elderly patients with acute myeloid leukemia

Acute myeloid leukemia (AML) predominantly affects the elderly, who often have a poor prognosis due to age-related comorbidities, adverse genetic features, and limited tolerance to standard therapies. This study aimed to evaluate real-world survival outcomes and prognostic factors in elderly patients with AML to improve clinical management. A retrospective analysis was conducted on 179 elderly AML patients across multiple centers over 6 years. Clinical features, bone marrow characteristics, vital status, and prognostic factors were analyzed. Kaplan–Meier was used to estimate overall survival, while univariate and multivariate regression analyzes identified prognostic factors. The median overall survival (mOS) of the cohort was 5.3 months. Patients in the chemotherapy group (n = 126) showed better mOS than those in the support therapy group (n = 53) (7.567 vs. 3 months; p<0.0001). Among chemotherapy patients, those treated with hypomethylating agents (HMAs) (n = 54) had better mOS compared to cytotoxic chemotherapy (n = 72) (10.17 vs. 4.1 months; p<0.0001). Within the HMA group, no significant difference in mOS was found between HMA monotherapy (n = 9) and HMA plus venetoclax (VEN) (n = 45) (9.117 vs. 10.17 months; p=0.3407). In patients eligible for intensive chemotherapy, the HMA group had a superior mOS than the cytotoxic chemotherapy group (9.4 vs. 3.933 months; p<0.0001). The top five mutations identified were NPM1 (29.59%), FT3-internal tandem duplication (ITD) (26.53%), DNMT3A (25.51%), IDH2 (20.41%), and CEBPA (14.29%), with DNMT3A-FLT3-ITD, NPM1-FLT3-ITD, and NPM1-DNMT3A showing significantly higher co-mutation frequencies than the other combinations. In addition, infection was the most frequent complication (60%). Elderly AML patients have poor mOS and a high burden of adverse genetic features. Chemotherapy, especially HMAs alone or combined with VEN, is associated with improved survival and better clinical outcomes compared to supportive care or intensive regimens. These findings provide real-world evidence to inform treatment strategies.