The Introduction of Hypomethylation Agents and Venetoclax into Standard Care for Older Patients with Acute Myeloid Leukemia: A Retrospective Real-World Analysis of Feasibility, Safety, and Effectiveness from an Outpatient Hematology Center.

Abstract

Introduction: The combination of venetoclax (VEN) and a hypomethylation agent (HMA) was introduced for the treatment of acute myeloid leukemia (AML) because it achieved significantly better results compared to HMA monotherapy (HMA-mono). Since introducing a new method into standard care is always a challenge, the goal of this retrospective analysis was to examine AML treatment before and after the approval of HMA-VEN at an office-based center for hematology and oncology (MVZ).

Methods: All newly diagnosed AML patients between January 2017 and April 2025 were fully documented and analyzed in an Excel database.

Results: In total, 65 patients were included; of these, 22 (mean age 59.6 years (y) (SD 15.35)), were admitted to the university hospital. Of the remaining 43 patients (mean age 79.4 y (SD 5.86)), 1 was treated with cytosine arabinoside (ARA-C), 7 with best supportive care (BSC), 15 with HMA-mono, and 20 with the HMA-VEN. The mean overall survival (OS) of the BSC, HMA-mono, and HMA-VEN was 19.6 (SD 15.04), 25.9 (SD 28.87), and 51.7 (SD 49.09) weeks, respectively. In the HMA-VEN cohort there was no tumor lysis syndrome, 11 (55%) patients had an infection, and fourteen (70%) required hospitalization during the first two treatment cycles.

Conclusion: HMA-VEN has been successfully integrated into standard care, which is primarily provided on an outpatient basis, and exhibits a safety and toxicity profile comparable to that of inpatient treatment. HMA-VEN is well tolerated subjectively in the subgroup of older patients and, in a historical comparison with the previous standard therapy, HMA-mono, or BSC, results in significantly longer overall survival without leading to an increase in complications or premature deaths.