Venetoclax combined with azacitidine in elderly acute myeloid leukemia: A retrospective comparison of 14-day vs 28-day dosing regimens

Abstract

However, this study has several limitations that must be acknowledged. First, the non-randomized allocation of treatment duration introduces potential selection bias, particularly as frailer patients were more likely to receive shorter therapeutic cycles, which may have confounded outcome assessments. Background: Although the standard 28-day venetoclax (VEN) regimen combined with azacitidine (AZA) improves outcomes in elderly patients with acute myeloid leukemia, emerging evidence suggests that shorter VEN cycles may maintain efficacy with enhanced safety. We retrospectively analyzed 90 treatment-naive elderly patients with acute myeloid leukemia receiving VEN + AZA (VA): 47 patients (14-day VEN) and 43 patients (28-day VEN). The outcomes included clinical remission rates, hematologic recovery, adverse events, and survival metrics. Both groups achieved comparable clinical remission rates (CRc: 57.4% vs 58.1%, P = .947). The 14-day cohort demonstrated significantly faster neutrophil recovery (median 12.5 vs 26 days, P < .01) and reduced febrile neutropenia (73.3% vs 90.9%, P < .05), with trends toward fewer grade ≥3 infections. At a median follow-up of 494 days, no significant differences in median overall survival (OS: 494 vs 578 days, HR = 1.17, 95%CI 0.64-2.14) or event-free survival (416 vs 454 days, HR = 1.09, 95%CI 0.61-1.96) were observed. A 14-day VA regimen showed antileukemic efficacy comparable to the 28-day protocol while mitigating myelosuppressive sequelae. This abbreviated approach may optimize tolerability in frail elderly patients who are ineligible for prolonged low-intensity chemotherapy. Prospective validation is warranted to refine risk-adapted dosing strategies.