Ivosidenib treatment in IDH-mutant WHO grade 4 astrocytomas: illustrative case
Year: 2026
Type: article
Study design: Case Report
Abstract: BACKGROUND: Isocitrate dehydrogenase (IDH) is a commonly mutated gene in gliomas. Although IDH-mutant WHO grade 4 astrocytomas tend to occur in younger patients and show an improved survival compared to IDH-wildtype, prognosis remains poor and treatment options are limited. Ivosidenib is a small-molecule inhibitor of IDH-1 that has shown promise for treating low-grade IDH-mutant glioma. However, little is known about its efficacy in IDH-mutant WHO grade 4 astrocytoma. OBSERVATIONS: The authors present the case of a 36-year-old male with a IDH-mutant WHO grade 4 astrocytoma. He was originally treated with resection, followed by radiation therapy and temozolomide. At the second recurrence, ivosidenib was started, after lomustine therapy had failed to control the disease. Radiological surveillance showed interval decrease in tumor size starting 2 months after initiation of ivosidenib. The patient remained neurologically intact, and no adverse effects were observed. LESSONS: Here the authors report the effects of the off-label use of ivosidenib. The INDIGO trial published evidence of efficacy in using an IDH inhibitor for low-grade gliomas. The role of these drugs in high-grade IDH-mutant gliomas is currently unknown. Further studies are needed to assess their impact on overall and progression-free survival. https://thejns.org/doi/10.3171/CASE25572.
DOI: 10.3171/case25572
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
OpenAlex ID: W7129228756
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