Real-World Outcomes of Venetoclax and Azacitidine in Hospitalized Patients With Acute Myeloid Leukemia: A Retrospective Study at a Tertiary Care Center.

Abstract

Venetoclax plus azacitidine improves survival in older or unfit patients with newly diagnosed acute myeloid leukemia (AML), as demonstrated in the VIALE-A trial. ² However, real-world data in hospitalized patients-particularly those with secondary AML or adverse-risk mutations-remain limited. We conducted a retrospective study of exclusively hospitalized patients with treatment-naïve AML who completed at least one full 28-day cycle of azacitidine and venetoclax at Baylor University Medical Center between October 2019 and October 2024, with the aim of evaluating outcomes at a clinically relevant treatment inflection point. Risk stratification was performed using ELN 2022 criteria, and safety was assessed using CTCAE v5.0. Fifty-six patients met inclusion criteria (median age 71 years); 51.8% had secondary AML, and 53.7% had adverse-risk disease. Among 43 evaluable patients, the composite complete remission (CRc) rate after one cycle was 55.8%. Median overall survival (OS) was 17.6 months. Patients with TP53 mutations had significantly shorter median OS (3.8 months) compared with those without TP53 mutations (20.2 months). Common grade ≥ 3 toxicities included leukopenia (67.8%) and febrile neutropenia (57.6%). By focusing on patients who completed an initial treatment cycle, this study provides conditional survival estimates that may inform decisions regarding continuation of therapy. Despite the predominance of secondary and adverse-risk AML in this cohort, OS was comparable to that observed in VIALE-A, supporting the effectiveness of azacitidine plus venetoclax in hospitalized AML patients ineligible for intensive chemotherapy.