Revumenib (menin inhibitor; KMT2A/NPM1 program) maintenance post-alloSCT shows 89% 2-year OS with low 1-year relapse

Revumenib (Revuforj) is an oral menin inhibitor that disrupts the menin–KMT2A interaction and is FDA-approved (since November 15, 2024) for R/R acute leukemia with KMT2A rearrangement (≥1 year old); here it was used as post–allogeneic SCT maintenance after pre-SCT response in KMT2Ar/NPM1mt/NUP98r AML (single-institution cohort; n=21; median age 20; 76% KMT2Ar; median 3 prior therapies; 57% prior SCT). Revumenib was started a median 2.8 months post-SCT and continued a median 6.7 months; with median follow-up 18.2 months, 1- and 2-year OS were both 89% and 1- and 2-year EFS were 84% and 73%, with 1-year cumulative incidence of relapse (CIR) 11% (and 5% death); in CR2+ (n=17), 2-year OS was 93% and 1-year CIR 13%—numerically better than the authors’ pre–menin inhibitor historical SCT cohort for the same genotypes (CR2+: 2-year OS 33%, 1-year CIR 46%). Safety was dominated by thrombocytopenia (86% any-grade; 43% grade ≥3), driving dose modifications in 48% and discontinuation in 10%; GVHD occurred in 14% (grade ≥3 5%), grade ≥3 infections in 10% (one grade 4 septic shock discontinuation), and no QTc prolongation was observed.