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December 8, 2025BloodOpen Access

Ivosidenib leads to durable responses in IDH1 mutated clonal cytopenias of undetermined significance: A phase II decentralized clinical trial

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Authors

KBKelly L. BoltonYMYazan F. MadanatASAbhay Singh

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Overview

Decentralized trial shows improved hematologic response in patients with IDH1-mutant clonal cytopenias, suggesting a viable treatment pathway.

Key Points

  • Overall, 82% of patients achieved a hematologic response with improvement in neutropenia observed in 88%.
  • Grade ≥3 adverse events occurred in 26% of patients treated with ivosidenib, including rare serious events.
  • A decentralized phase II clinical trial was conducted, monitoring patients for 8 weeks of treatment effectiveness.
  • Findings support the potential of ivosidenib to modify the progression of IDH1-mutant clonal cytopenias.

Cite This Study

Bolton et al. (2025) studied this question.

synapsesocial.com/papers/69362f3a4fa91c937236d40fhttps://doi.org/10.1182/blood-2025-635
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Real-world experience with IDH inhibitors in Acute Myeloid Leukemia: A german multicenter analysis with a focus on differentiation syndrome2025
  2. 2Clinical and translational study of ivosidenib plus nivolumab in advanced solid tumors harboring IDH1 mutations2025 · 5 citations
  3. 3Safety and efficacy of ivosidenib in the treatment of isocitrate dehydrogenase 1 mutant cholangiocarcinoma and acute myeloid leukemia: a systematic review and meta-analysis2025
  4. 4Ivosidenib for IDH1‐Mutant Intrahepatic Cholangiocarcinoma: Insights From a Multicenter Real‐World Study2025 · 4 citations
  5. 5Trial in progress – a phase 1b trial of ivosidenib combined with ruxolitinib in IDH1-mutated advanced myeloproliferative neoplasms2025