Abstract 5960: Outcomes of acute myeloid leukemia treated with a hypomethylating agent and venetoclax
Authors: Markie Zimmer, Brigid Jacob, Mikayla Spica, Sunita Ghosh, Vrushali Dabak
Journal: Cancer Research (2025)

Abstract Introduction: VIALE-A confirmed the benefit of azacitidine (AZA) and venetoclax (VEN) compared to AZA alone in previously untreated patients with AML who were not eligible for intensive induction chemotherapy. Incorporation of this regimen into standard practice has varied across and within healthcare systems due to adjustments in regimen and supportive care use. Methods: This was a retrospective, single institution analysis of adult patients greater than 18 years of age with AML, treatment related AML (t-AML) or MDS/AML treated with a hypomethylating agent (HMA) (azacitidine or decitabine) and VEN between January 2017 and January 2024. We sought to investigate overall survival (OS) and to explore the trends of regimen and prophylaxis dosing, the use of allogeneic stem cell transplant and outcomes of relapsed and refractory disease. Results: Thirty patients received VEN and HMA between January 2017 and January 2024. The median age was 72 years. Ten patients were female and 20 were male. Twenty-four patients were Caucasian, 5 were African American and one patient’s race was not recorded. Twenty-three patients were diagnosed with AML, 5 t-AML and 2 MDS/AML. Risk stratifying patients by ELN 2022 criteria, 5 (16.7%) were favorable, 6 (20%) were intermediate and 17 (56.7%) were adverse. Within the adverse risk group, 6 had mutated TP53. Patients completed a median of 3 cycles. The mean duration of VEN decreased throughout each cycle, from 21.60 days in cycle 1, to 15.38 in cycle 2, to 8.62 in cycle 3 and 8.10 in cycle 4. The median number of days between the starting of subsequent cycles generally increased (34.5 days from cycle 1 to cycle 2; 39 days from cycle 2 to cycle 3; 42 days from cycle 3 to cycle 4). Twenty-one patients received the treatment in the front-line setting, 7 as salvage therapy and 2 as salvage after allo-HSCT. Three patients proceeded to allo-HSCT a median of 179 days after initial diagnosis. Of all 30 patients, 9 (30%) were in remission, 20 (66.7%) had active disease and one patient’s disease status was unknown at the time of data collection. Mean overall survival was 22.67 months (5.92-39.42, 95% CI, p=0.346). Caucasians had greater than 6 times OS than African Americans, (26.12 months, 10.42-41.82, 95% CI versus 4.44 months, 0-10.85, 95% CI, p=0.015). Frontline use of the regimen resulted in a numerically significant difference in OS compared to salvage use (22.67 versus 4.53 months, p=0.200). Conclusions: In this retrospective analysis of VEN and HMA in patients with AML treated in an urban setting, we found that younger patients, Caucasians, favorable risk disease by ELN 2022 criteria and frontline use resulted in the highest OS. Overall, patients received decreasing number of days of VEN each cycle and had increasing number of days between cycles, suggesting significant treatment reduction and delays, however without compromise to overall survival. Citation Format: Markie Zimmer, Brigid Jacob, Mikayla Spica, Sunita Ghosh, Vrushali Dabak. Outcomes of acute myeloid leukemia treated with a hypomethylating agent and venetoclax [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2025; Part 1 (Regular Abstracts); 2025 Apr 25-30; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2025;85(8_Suppl_1):Abstract nr 5960.