ASH 2023 | Investigating ivosidenib for reducing the risk of CCUS progression to hematological malignancy

Myeloid malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), exist on a continuum and often progress from an entity termed clonal cytopenia of undetermined significance (CCUS). Patients with CCUS are at high risk of progression to hematological malignancies, with ~95% of these patients progressing over a 10-year period. Giulia Petrone, MD, MBBS, Washington University Medical School, St. Louis, MO, discusses a trial-in-progress (NCT05030441) enrolling patients with IDH1-mutant CCUS and treating them with the IDH1 inhibitor ivosidenib with the aim of improving cytopenia and reducing risk of progression to hematological malignancy. The innovative decentralized study design means that patients in rural areas can enroll in the trial, increasing the sample size and demonstrating how access to treatment can be improved. Dr Petrone hopes that the findings of this study will aid in identifying and treating patients with this entity, eventually leading to a decrease in the prevalence of MDS and AML. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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