Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome.

ABSTRACT:

Abstract

Olutasidenib, a potent, selective, oral small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (mIDH1), is US Food and Drug Administration approved for mIDH1 relapsed/refractory (R/R) acute myeloid leukemia based on results from the pivotal cohort of a multiarm phase 1/2 trial that also enrolled patients with myelodysplastic syndrome (MDS). We report pooled data evaluating olutasidenib as monotherapy or combined with azacitidine in R/R and treatment-naïve (TN) higher-risk mIDH1 MDS. Twenty-two patients (median age, 74 years; 59% male) with intermediate- to very high-risk MDS (monotherapy, n = 6 [4 R/R and 2 TN]; combination, n = 16 [11 R/R and 5 TN]) were analyzed. The most frequent adverse events were fatigue and cytopenias. Differentiation syndrome occurred in 3 p

SUBMITTER: Cortes JE 

PROVIDER: S-EPMC12552956 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature