AML-297: Ten-Day Decitabine with Venetoclax vs Intensive Chemotherapy in Relapsed or Refractory Acute Myeloid Leukemia: A Propensity Score-Matched Analysis

Abstract

Context: Relapsed/refractory (R/R) acute myeloid leukemia (AML) has poor outcomes. While lower-intensity venetoclax-containing regimens are standard for older/unfit patients with newly diagnosed AML, it is unknown how such regimens compare to intensive chemotherapy (IC) for R/R AML. Objective: To compare outcomes of R/R AML treated with 10-day decitabine and venetoclax (DEC10-VEN; NCT03404193) vs IC-based regimens Design: This was a retrospective study. Propensity scores derived from baseline characteristics were used to match patients treated with DEC10-VEN and IC to minimize bias. Patients and Setting: Sixty-five patients in the DEC10-VEN cohort were matched to 130 IC recipients treated at the University of Texas MD Anderson Cancer Center. The median ages for the DEC10-VEN and IC groups were 64 and 58 years, respectively, and baseline characteristics were balanced between the two cohorts. Interventions: DEC10-VEN comprised of decitabine for 10-days and venetoclax 400 mg daily (DiNardo et al. Lancet Haematol. 2020.) IC-based regimens included idarubicin with cytarabine (IA), with or without cladribine (CLIA), clofarabine (CIA), or fludarabine (FIA or FLAG-IDA), with or without additional agents. Main Outcome Measures: Response rates per ELN 2017 criteria, rate of negative MRD by flow cytometry, early mortality, safety, event-free survival (EFS), and overall survival (OS). Results: DEC10-VEN conferred significantly higher responses compared to IC, including higher overall response rate (60% vs 36%, odds ratio [OR]: 3.28, p Conclusions: DEC10-VEN represents an appropriate salvage therapy and may offer better responses and survival compared to IC in adults with R/R AML. Funding: NCI CCSG CA016672, R01CA235622.