Objective:

To evaluate whether glucagon-like peptide 1 receptor agonist (GLP-1 RA) therapy provides clinical benefits in the management of idiopathic intracranial hypertension (IIH) compared with conventional treatments.

Background:

Current therapies for IIH, including acetazolamide, topiramate, and weight management strategies, are limited by side effects and incomplete efficacy. GLP-1 RAs, widely used for weight loss and metabolic regulation, have emerged as potential agents for IIH treatment, but evidence remains limited.

Design/Methods:

A retrospective cohort study was performed using electronic health records from the TriNetX US Collaborative Network (2005-2024). Adults aged 18 years or older with an IIH diagnosis were included. Patients initiating GLP-1 RA therapy within six months of diagnosis formed the exposure group, while controls received conventional therapies without GLP-1 RA use. Outcomes were assessed over a one-year follow-up. Propensity score matching was applied to balance baseline characteristics, and risk ratios (RRs) with 95% confidence intervals (CIs) were calculated.

Results:

Among 44,373 patients with IIH, 603 received GLP-1 RAs and 43,770 did not. After matching, 555 patients remained in each group. Compared with controls, GLP-1 RA use was associated with reduced medication reliance (RR 0.53; 95% CI 0.46-0.61), headaches (RR 0.45; 95% CI 0.35-0.58), visual disturbances or blindness (RR 0.60; 95% CI 0.41-0.88), and papilledema (RR 0.19; 95% CI 0.10-0.34). Procedures (RR 0.44; 95% CI 0.30-0.63) and mortality (RR 0.36; 95% CI 0.18-0.73) were also lower. No significant differences in BMI were observed at one year. Findings were consistent across BMI subgroups. Bariatric surgery was associated with greater weight loss, but GLP-1 RA therapy was associated with better outcomes.

Conclusions:

GLP-1 RA therapy was associated with substantial improvements in clinical outcomes and reduced need for interventions in IIH. These results support GLP-1 RAs as a promising therapeutic option, meriting confirmation through prospective studies.