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This combination therapy consists of ivosidenib, a small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), and Onureg (oral azacitidine), a hypomethylating agent. It is being investigated by the French Innovative Leukemia Organisation (FILO) as a maintenance treatment for patients aged 55 and older with IDH1-mutated acute myeloid leukemia (AML) who have achieved complete remission following intensive chemotherapy. Ivosidenib works by reducing levels of the oncometabolite 2-hydroxyglutarate (2-HG) and promoting myeloid differentiation, while oral azacitidine acts as a DNA methyltransferase inhibitor to induce DNA hypomethylation and restore normal gene expression. The combination aims to improve relapse-free survival in this high-risk patient population.
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