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December 8, 2025BloodOpen Access

Real-world experience with IDH inhibitors in Acute Myeloid Leukemia: A german multicenter analysis with a focus on differentiation syndrome

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Authors

TLTim LohmannHDHartmut DöhnerTSTim Sauer

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Overview

Multicenter study shows adverse events including febrile neutropenia and differentiation syndrome in AML patients treated with IDH inhibitors.

Key Points

  • To assess the clinical activity of IDH inhibitors in acute myeloid leukemia, focusing on differentiation syndrome.
  • Conducted a retrospective multicenter study across eleven German tertiary care centers.
  • Included AML patients treated with enasidenib or ivosidenib outside of clinical trials.
  • Evaluated responses based on ELN 2022 criteria and performed next-generation sequencing where available.
  • 56 patients were analyzed, with most carrying an IDH1 mutation and treated predominantly with ivosidenib.
  • Clinically relevant differentiation syndrome occurred in 29% of patients, primarily those treated with ivosidenib.
  • Febrile neutropenia was observed in 52% of patients with IDH-mutant AML.

Cite This Study

Lohmann et al. (2025) studied this question.

synapsesocial.com/papers/693624ce4fa91c937236cf8fhttps://doi.org/10.1182/blood-2025-2846
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Also Consider

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

  1. 1Ivosidenib leads to durable responses in IDH1 mutated clonal cytopenias of undetermined significance: A phase II decentralized clinical trial2025 · 2 citations
  2. 2Ivosidenib combined with venetoclax and azacitidine for the treatment of newly diagnosed IDH1-mutant Acute Myeloid Leukemia: A prospective, two cohorts, multicenter study2025
  3. 3A phase II study of IDH1 inhibition with ivosidenib as maintenance therapy after ivosidenib, azacitidine, and venetoclax for Acute Myeloid Leukemia: Trial in progress2025
  4. 4IDH mutant MDS: Proposal for disease subset recognition based on molecular and clinical features, and the availability of targeted therapies2025
  5. 5P12.23.B IDH INHIBITION AT RELAPSE?2025