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Ivosidenib Monotherapy Shows Promise in IDH1 Mutated Myelodysplastic Neoplasm/Syndrome: A Phase 2 Study

MedXY Editorial Team•Aug 28, 2026•Hematology-Oncology
targeted therapyivosidenibIDH1 Mutationmyelodysplastic syndrome

Highlight

– Ivosidenib (IVO), an oral inhibitor targeting mutant IDH1, was studied as monotherapy in patients with IDH1 mutated myelodysplastic neoplasm/syndrome (MDS).

– The phase 2 trial enrolled 48 patients across three cohorts: high-risk relapsed/refractory post-Azacitidine, treatment-naïve high-risk, and low-risk patients refractory to erythropoiesis-stimulating agents (ESAs).

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