GLP-1 Receptor Agonists May Improve Outcomes in Idiopathic Intracranial Hypertension

Background Idiopathic intracranial hypertension (IIH) remains a challenging condition to manage, with current treatment options such as acetazolamide, topiramate, and lifestyle modifications limited by issues of efficacy, safety, and sustainability. Glucagon‐like peptide‐1 receptor agonists (GLP‐1 RAs), widely used for weight loss and metabolic regulation, may represent a novel therapeutic strategy for IIH. Methods We conducted a retrospective cohort study using the TriNetX US Collaborative Network, encompassing data from 69 healthcare organizations between 2005 and 2024. Adults (≥18 years) diagnosed with IIH were eligible. Among 43,875 identified patients, 528 initiated GLP‐1 RA therapy and 43,347 received conventional treatments without GLP‐1 RA exposure. Propensity score matching (PSM) created two balanced cohorts of 499 patients each. Exposure was defined as GLP‐1 RA initiation within six months of IIH diagnosis. Outcomes assessed over one year included medication use, symptom burden, procedural interventions, and mortality, expressed as risk ratios (RR) with 95% confidence intervals (CI). Results After PSM, GLP‐1 RA use was associated with significantly lower medication use (29.5% vs. 59.3%; RR = 0.497, 95% CI 0.426‐0.579; p<0.001) and reduced prevalence of symptoms such as headache (12.8% vs. 26.7%; RR = 0.481, 95% CI 0.367‐0.631; p<0.001) and visual disturbances (6.4% vs. 16.6%; RR = 0.386, 95% CI 0.261‐0.569; p<0.001). Procedural interventions were less common in the GLP‐1 RA group (6.8% vs. 19.0%; RR = 0.358, 95% CI 0.247‐0.519; p<0.001), including lower rates of bariatric surgery (5.4% vs. 11.2%; RR = 0.482, 95% CI 0.310‐0.750; p = 0.001). Mortality was lower in the GLP‐1 RA group (0.2‐2.0% vs. 3.8%), though statistical comparisons were limited by small sample sizes. Conclusions GLP‐1 RA therapy in IIH is associated with significant reductions in medication use, symptoms, and procedural interventions, suggesting its potential as an effective management strategy. Further prospective studies are warranted to confirm these findings.