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ASH 2021 | IDIOME: ivosidenib in IDH1 mutant MDS

Ivosidenib, a small molecule inhibitor of mutant IDH1, is FDA approved for the treatment of patients with relapsed/refractory acute myeloid leukemia (AML) with an IDH1 mutation. To investigate the efficacy of ivosidenib in IDH1-mutant myelodysplastic syndrome (MDS), the Phase II IDIOME study was established (NCT03503409), to which 32 patients were enrolled at the point of data cut-off. Thomas Cluzeau, MD, PhD, Central University Hospital of Nice, Nice, France, discusses the preliminary trial findings. Participants were recruited to three cohorts: those with higher risk MDS having failed azacitidine (cohort A), untreated higher risk MDS without life threatening cytopenias (cohort B), and lower risk MDS having failed EPO (cohort C). The overall response rate (ORR) of the 26 evaluable patients was 69%, with the highest response rates achieved in cohort B (91% ORR). After a medium of 9.1 months of follow up, the medium duration of response was 7.4 months. The promising preliminary safety and efficacy data gathered to date support the continuation of the IDIOME study. This interview took place at during 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.