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GLP-1 RAs May Offer Novel Approach for Idiopathic Intracranial Hypertension Management

Patients with idiopathic intracranial hypertension (IIH) treated with glucagon-like peptide 1 receptor agonists (GLP-1 RAs) had lower medication use, fewer symptoms, and a reduced need for procedural interventions over 1 year compared with propensity score–matched patients treated with conventional IIH therapies, according to study results published in JAMA Neurology.

“Bariatric surgery was associated with greater weight loss, but GLP-1 RA therapy was associated with better outcomes,” wrote Georgios S. Sioutas, MD, and coauthors from the neurosurgery department at Virginia Commonwealth University Health System, Richmond, Virginia.

With current IIH treatment options limited by concerns over efficacy, safety, and sustainability, the retrospective cohort study focused on outcomes for patients with IIH treated with GLP-1 RA therapy compared with conventional approaches. Researchers assessed data from the TriNetX US Collaborative Network for adults with IIH treated at 67 health care organizations across the United States between 2005 and 2024.

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After propensity score matching, 555 patients who initiated GLP-1 RA therapy within 6 months of IIH diagnosis were compared with 555 patients managed with conventional treatments, such as acetazolamide, topiramate, and dietary counseling, with no GLP-1 RA exposure.

According to the study, GLP-1 RA users had lower use of non-GLP-1 RA medication (researchers reported a 0.53 risk ratio [RR]), as well as reduced headaches (0.45 RR), visual disturbances or blindness (0.60 RR), and papilledema (0.19 RR), over 1 year of follow-up. The GLP-1 RA group also had lower need for procedures (0.44 RR) and lower mortality (0.36 RR) over 1 year compared with the conventional treatment group.

The 2 groups did not significantly differ in mean body mass index (BMI) at follow-up, which was 40.6 in the GLP-1 RA group vs 39.5 in the conventional treatment group.

Researchers found similar results in a sensitivity analysis stratified by BMI (≥40 vs <40).

“GLP-1 RA therapy may offer an alternative management strategy for IIH,” the authors wrote, “warranting further prospective evaluation.”

 

Reference

Sioutas GS, Mualem W, Reavey-Cantwell J, Rivet DJ 2nd. GLP-1 receptor agonists in idiopathic intracranial hypertension. JAMA Neurol. Published online July 14, 2025. doi:10.1001/jamaneurol.2025.2020