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

Abstract Background 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. Methods 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 ). Results 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). Conclusions 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.