Outcomes by Best Response With Hypomethylating Agent Plus Venetoclax in Adults with Previously Untreated Acute Myeloid Leukemia

Abstract

Introduction: We aimed to compare outcomes of patients with AML treated with frontline hypomethylating agent and venetoclax (HMA+Ven) who achieved complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), or morphologic leukemia-free state (MLFS) as defined by ELN 2022. Methods: Patients with AML seen at Moffitt Cancer Center between 2018 and 2022 and treated with HMA+Ven were retrospectively evaluated. Results: About 120 patients achieved blast clearance with best response of CR in 52 (43.3%), CRh in 22 (18.3%), CRi in 31 (25.8%) and MLFS in 15 (12.5%) patients. Greater proportion of patients with MLFS had a prior myeloid malignancy (p=0.003) and were treated with prior HMA (p<0.001). Patients that achieved MLFS as their best response had inferior OS compared to the CR/CRh/CRi cohort (8 months vs 27 months; p<0.001). RFS was also worse for the MLFS cohort. Conclusion: To the best of our knowledge, this is the largest study analyzing differences in outcomes of AML patients treated with HMA+Ven based on best response. We noted that prior myeloid malignancy and use of HMA led to more MLFS as best response compared to CR/CRi. The OS and RFS were inferior for MLFS cohort.