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.

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.