GLP-1 Receptor Agonists Linked to Better Outcomes in Idiopathic Intracranial Hypertension Than Standard Therapy

Results from a large retrospective study published in JAMA Neurology suggest that glucagon-like peptide-1 receptor agonist (GLP-1 RA) therapy may offer benefits for patients with idiopathic intracranial hypertension (IIH). Compared with conventional therapies, GLP-1 RA use was associated with reductions in medication use, headaches, papilledema, and the need for invasive procedures. These findings highlight the potential of GLP-1 RAs as an alternative management strategy for people with IIH.

Drawing from the TriNetX US Collaborative Network, researchers analyzed the electronic health records of 44,373 adults diagnosed with IIH between 2005 to 2024. After propensity score matching, 555 patients who initiated GLP-1 RA therapy within 6 months of diagnosis were compared with 555 patients treated with standard approaches such as acetazolamide, topiramate, and dietary counseling. Outcomes were assessed over a 1-year follow-up period.

Key findings include:

• Participants treated with GLP-1RA therapy were less likely to require the use of medications (n=165; 29.7%) than those treated with standard therapy (n=313; 56.4%) (P<.001).
• GLP-1RA therapy was associated with reduced rates of headache (n=65 [12.3%] vs n=152[27.4%]; P<.001), visual disturbances/blindness (n=39 [7.0%] vs n=65[11.7%]; P=.007), and papilledema (n=12 [2.2%] vs n=64[11.5%]; P<.001).
• Those who received GLP-1RA therapy required fewer procedural interventions (n=38; 6.8%) than those who received standard therapy (n=87; 15.7%) (P<.001) including lower rates of CSF shunt placement (P=.002)
• Mortality was lower in the GLP-1 RA group (n=1 to 10; ≤2.0%) vs the standard therapy group (n=28; 5.0%) (P=.003).

Source: Sioutas GS, Mualem W, Reavey-Cantwell J, Rivet DJ. GLP-1 Receptor Agonists in Idiopathic Intracranial Hypertension. JAMA Neurol. 2025;82(9):887–894. doi:10.1001/jamaneurol.2025.2020