Prognostic value of Day 14 measurable residual disease in acute myeloid leukemia treated with venetoclax and azacitidine.

Abstract

Venetoclax (VEN) in combination with azacitidine (AZA) (VEN-AZA) is used to treat acute myeloid leukemia (AML) in patients who are not candidates for intensive chemotherapy but research on prognostic factors remains limited. Measurable residual disease (MRD) by multiparametric flow cytometry in AML is important but there is limited evidence of the clinical utility of monitoring MRD in patients treated with VEN-AZA. Herein, a total of 75 patients newly diagnosed with AML treated with VEN-AZA were retrospectively analyzed to examine the role and timing of MRD to predict survival. MRD enabled the categorization of patients into two groups: Day 14 MRD, >1% (MRD^14-pos); and Day 14 MRD, ≤1% (MRD^14-neg). Of the 75 patients, 31 (41.3%) had MRD^14-neg, whereas 30 (40.0%) had not achieved complete remission (CR) after induction. MRD^14-neg was associated with improved overall survival (OS) (p = .024) and event-free survival (EFS) (p = .044). In addition, MRD^14-neg (p = .002 for both OS and EFS), CSF3R negative (CSF3R^neg) (p < .001 for both OS and EFS), and transplantation (p = .005 for OS; p = .007 for EFS) were associated with improved survival outcomes. Further subgroup analysis revealed that MRD^14-pos patients who underwent transplantation showed a trend toward longer OS and EFS (p < .001 for both). Results in the MRD^14-neg group were better than in the MRD^14-pos group, and the prognosis for patients with AML was better when there was CSF3R^neg and transplantation. Additionally, for patients with AML with MRD^14-pos, consolidation with transplantation may increase survival.