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Background <p>Idiopathic intracranial hypertension (IIH) is a debilitating neurologic condition marked by elevated intracranial pressure (ICP), typically affecting obese women of reproductive age. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs), widely used for obesity and diabetes, have recently emerged as potential therapeutic agents for IIH.</p> Objective <p>To systematically evaluate the efficacy and safety of GLP-1 RAs in IIH, based on clinical studies reporting outcomes related to ICP, headache, vision, cognition, weight loss, and safety.</p> Methods <p>A systematic search was conducted across MEDLINE, Scopus, and Web of Science up to June 15, 2025, using PRISMA 2020 guidelines. Studies involving adult IIH patients treated with GLP-1 RAs and reporting relevant clinical outcomes were included. Data extraction and bias assessment were performed independently by multiple reviewers.</p> Results <p>Nine clinical studies (one RCT, eight observational studies) with IIH patients receiving GLP-1 RAs were included. GLP-1 RA use was consistently associated with improvements in headache frequency, weight reduction, and reduced need for acetazolamide or surgical interventions. In a small RCT, exenatide significantly lowered ICP within hours of administration. Several studies reported papilledema resolution and a lower risk of visual deterioration. Cognitive function either improved or remained stable. Adverse effects were mostly mild gastrointestinal symptoms; no serious safety concerns emerged. Interpretation is limited by the predominance of potentially overlapped retrospective data and minimal randomized evidence.</p> Conclusion <p>GLP-1 RAs show promise as a disease-modifying therapy for IIH, with potential benefits beyond weight loss. The consistency of findings across multiple studies and data sources provides justification of a shift in research focus to conduct well-powered phase 3 randomized controlled trials to validate these preliminary findings and define optimal treatment strategies.</p>

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Use of glucagon-like peptide-1 receptor agonists in idiopathic intracranial hypertension : a systematic review

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年10月7日

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  • Julien Ognard,
  • Sevda Alipour Khabir,
  • Sherief Ghozy,
  • Gerard El Hajj,
  • Kevin M. Kallmes,
  • John J. Chen,
  • Ramanathan Kadirvel,
  • David F. Kallmes,
  • Waleed Brinjikji

摘要

Background

Idiopathic intracranial hypertension (IIH) is a debilitating neurologic condition marked by elevated intracranial pressure (ICP), typically affecting obese women of reproductive age. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs), widely used for obesity and diabetes, have recently emerged as potential therapeutic agents for IIH.

Objective

To systematically evaluate the efficacy and safety of GLP-1 RAs in IIH, based on clinical studies reporting outcomes related to ICP, headache, vision, cognition, weight loss, and safety.

Methods

A systematic search was conducted across MEDLINE, Scopus, and Web of Science up to June 15, 2025, using PRISMA 2020 guidelines. Studies involving adult IIH patients treated with GLP-1 RAs and reporting relevant clinical outcomes were included. Data extraction and bias assessment were performed independently by multiple reviewers.

Results

Nine clinical studies (one RCT, eight observational studies) with IIH patients receiving GLP-1 RAs were included. GLP-1 RA use was consistently associated with improvements in headache frequency, weight reduction, and reduced need for acetazolamide or surgical interventions. In a small RCT, exenatide significantly lowered ICP within hours of administration. Several studies reported papilledema resolution and a lower risk of visual deterioration. Cognitive function either improved or remained stable. Adverse effects were mostly mild gastrointestinal symptoms; no serious safety concerns emerged. Interpretation is limited by the predominance of potentially overlapped retrospective data and minimal randomized evidence.

Conclusion

GLP-1 RAs show promise as a disease-modifying therapy for IIH, with potential benefits beyond weight loss. The consistency of findings across multiple studies and data sources provides justification of a shift in research focus to conduct well-powered phase 3 randomized controlled trials to validate these preliminary findings and define optimal treatment strategies.