Evaluation of treatment outcomes for AML patients ineligible for intensive chemotherapy before and after the implementation of VEN/AZA - a retrospective cohort study.

OBJECTIVES: Acute myeloid leukemia (AML) predominantly affects older patients, many of whom are ineligible for intensive chemotherapy. Although the VIALE-A trial demonstrated improved survival with azacitidine plus venetoclax, its strict eligibility criteria may limit generalizability to routine clinical practice. We therefore evaluated real-world outcomes at our institution. METHODS: We conducted a retrospective single-centre cohort study of adult AML patients ineligible for intensive chemotherapy. Patients received azacitidine monotherapy (2014-2018) or azacitidine plus venetoclax (2019-2023). Overall survival (OS) and progression-free survival (PFS) were analyzed using Kaplan-Meier estimates and log-rank tests. VIALE-A eligibility was assessed retrospectively. RESULTS: 21 patients met the inclusion criteria: 8 received azacitidine alone and 13 azacitidine plus venetoclax. Median age was 79 years. Patients receiving azacitidine plus venetoclax completed more treatment cycles (median 7 vs. 4). Median OS was 12.6 vs. 9.1 months with azacitidine alone (p=0.416). Median PFS was 10.0 vs. 8.1 months, respectively (p=0.707). Overall, only 66 % of patients fulfilled the VIALE-A eligibility criteria. CONCLUSIONS: Azacitidine plus venetoclax was associated with numerically longer OS and PFS, although differences were not statistically significant. Only two-thirds of patients would have met VIALE-A eligibility criteria, highlighting the challenges of translating trial outcomes into routine practice. Larger multicentre studies are warranted.