BACKGROUND: Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are gaining popularity as multipurpose treatment modalities with an emerging role in the treatment of idiopathic intracranial hypertension (IIH). This meta-analysis aimed to summarize the efficacy and safety of GLP-1 RAs in comparison to conventional management.
METHODS: MEDLINE and Cochrane databases were searched from inception through August 2025 using keywords related to "idiopathic intracranial hypertension", "papilledema", and "GLP-1 receptor agonist". All studies comparing adult IIH patients receiving GLP-1 RAs to those receiving conventional management were eligible for inclusion. Studies including patients having received any prior treatment for IIH were excluded. Of the initially retrieved 35 articles, 9 studies were included in our meta-analysis. The main outcome measures included change in body mass index (BMI), intracranial pressure (ICP), headache-related parameters, and ophthalmologic parameters. Safety outcomes included mortality risk, risk of refractory IIH (defined as needing surgical intervention), and other adverse events.
RESULTS: Nine studies comprising 13,257 participants were included in this meta-analysis. The results suggested that GLP-1 RAs confer a beneficial treatment effect with respect to risk of headache (RR: 0.73; 95 % CI: 0.61-0.86), papilledema (RR: 0.38; 95 % CI: 0.25-0.56), visual worsening (RR: 0.51; 95 % CI: 0.37-0.68), mortality (RR: 0.20; 95 % CI: 0.07-0.59), and risk of refractory IIH (RR: 0.76; 95 % CI: 0.65-0.89)).
CONCLUSIONS: Our meta-analysis demonstrates that GLP-1 RAs are more effective than conventional treatment options for managing IIH, with particular utility in preventing disease progression and surgical intervention. Future well-powered RCTs are necessitated to validate our findings.
