Venetoclax and azacitidine versus CAG for unfit patients with newly diagnosed acute myeloid leukemia: A propensity score-matched analysis.

This study evaluated the clinical efficacy of venetoclax and azacitidine (VEN+AZA) versus CAG (cytarabine, aclarubicin, and granulocyte colony-stimulating factor) for newly diagnosed adult acute myeloid leukemia (AML) unfit patients. There were 248 patients received VEN+AZA and 104 patients received CAG. A PSM (Propensity Score-Matching) was conducted using the nearest neighbor method in a 1:2 ratio to minimize bias. As a result, 170 patients in the VEN+AZA cohort were matched with 85 patients in the CAG cohort. In propensity-matched group, VEN+AZA had higher CRc rate than the CAG (all cohort, 67.9 % vs 55.5 %, P = 0.04), especially for age ≥ 60 y (OR=1.085, P = 0.04), secondary AML(OR=1.802, P = 0.028), ELN intermediate-adverse karyotype(OR=1.33, P = 0.045), DNMT3A mutation (OR=1.32, P = 0.047) and IDH1/2 mutation (OR=1.952, P = 0.049), but patients with RUNX1::RUNX1T1 (OR=0.25, P = 0.044) favored CAG in CRc rate. The 3-year OS and EFS for VEN+AZA group and CAG group were 49.8 % VS. 49.8 % (P = 0.81) and 34.3 % VS. 31.7 % (P = 0.20). Patients with age ≥ 60 y (EFS, P = 0.032), ECOG≥ 2 (EFS, P = 0.047), secondary AML (OS, P = 0.015; EFS, P = 0.039), ELN intermediate-adverse karyotype (OS, P = 0.034; EFS, P = 0.044), RUNX1 mutation (OS, P = 0.003; EFS, P = 0.003) and IDH1/2 mutation (EFS, P = 0.039) showed a preference for VEN+AZA regarding OS, and patients with SRSF2 mutation favored CAG in OS (P = 0.031).