Abstract:
Background Despite achieving minimal residual disease (MRD) negativity through consolidation therapies such as chemotherapy or autologous stem cell transplantation (ASCT), patients with intermediate-risk acute myeloid leukemia (AML) still face a high cumulative incidence of relapse (CIR) of 30% to 50% within two years. Current maintenance therapies struggle to balance efficacy and safety. Objective To investigate the long-term efficacy and safety of genetically modified dendritic cell (gmDC) vaccine combined with cytokine-induced killer (CIK) cells as maintenance therapy in patients with intermediate-risk AML. Methods This retrospective cohort study recruited intermediate-risk AML patients treated at our hospital from January 2013 to December 2023. All enrolled patients achieved MRD negativity after consolidation chemotherapy. Based on treatment, they were classified into the gmDC vaccine combined with CIK maintenance group (gmDC group) and the routine follow-up observation group (control group). The primary endpoint was overall survival (OS). Key secondary endpoints included progression‑free survival(PFS) and CIR. The secondary endpoint was safety. Results A total of 23 patients with intermediate‑risk AML were enrolled in this study, including 13 cases in the gmDC group and 10 cases in the control group. The median ages of the two groups were 34(range, 16 ~ 83) years and 43(range, 19 ~ 62) years, respectively, and the proportions of male patients were 46.2% and 40.0%, respectively, with no statistically significant differences (all P>0.05). Baseline characteristics were balanced between the two groups except for ASCT (gmDC group: 53.8% vs control group: 0, P=0.007). After a median follow-up of 29 months, the gmDC group demonstrated significantly higher 2-year OS (100.0% vs 40.0%, P=0.001) and 2-year PFS (84.6% vs 20.0%, P=0.001), and a significantly lower 2- year CIR (15.4% vs 80.0%, P=0.001) compared with the control group. Multivariable analysis showed that the hazard ratio (HR) for OS was 0.108 (95% CI: 0.013 - 0.937, P=0.043); the HR PFS was 0.151 (95% CI: 0.028 - 0.811, P=0.027); and the subdistribution hazard ratio (sHR) for CIR was 0.108 (95% CI: 0.025 - 0.456, P=0.002). Only grade 1 adverse events occurred in the gmDC group, with no grade ≥ 2 events.Conclusion In maintenance therapy for intermediate-risk AML, the gmDC vaccine combined with CIK shows potential to improve survival and reduce relapse risk, with a favorable safety profile. Given the retrospective design and the imbalance in ASCT rates between the two groups, this conclusion warrants further validation in prospective randomized controlled trials.