Potential therapeutic role of GLP-1 receptor agonists in Idiopathic intracranial hypertension: a systematic review and meta-analysis

Mar 4, 2026Neurological research

Possible treatment use of GLP-1 receptor activators for unexplained high pressure in the brain: a review and combined analysis

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Abstract

GLP-1 receptor agonists significantly reduced refractory idiopathic intracranial hypertension incidence by 60%.

The risk of papilledema was reduced by 60% with GLP-1 receptor agonist treatment.
Visual disturbances and blindness were also reduced by 49%.
No significant changes were observed in objective visual measures, such as visual acuity or retinal nerve fiber layer thickness.
GLP-1 receptor agonists were associated with a reduction in headache incidence and fewer monthly headache days.
Changes in body mass index were not statistically significant.

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BACKGROUND: Idiopathic intracranial hypertension (IIH) is a neurological disorder characterized by elevated intracranial pressure (ICP) , often resulting in headaches and visual impairment. Glucagon-like peptide-1 receptor agonists (GLP-1RAs), commonly used in the treatment of metabolic disorders, have emerged as potential therapeutic strategies for IIH.

METHODS: We systematically searched PubMed, Embase, Web of Science, Scopus, and the Cochrane Library up to July 2025, following PRISMA guidelines. Primary outcome was incidence of refractory IIH; secondary outcomes included visual function, headache-related measures, and BMI. Random-effects models were used to pool relative risks (RRs) and standardized mean differences (SMDs).

RESULTS: Seven studies involving 11,973 participants were included. GLP-1RAs significantly reduced refractory IIH incidence (and the risk of papilledema (RR = 0.40, 95% CI: 0.22-0.72), as well as visual disturbances and blindness (RR = 0.51, 95% CI: 0.28-0.95). However, no significant improvements were observed in objective visual parameters, including visual acuity, visual field, or peripapillary retinal nerve fiber layer (pRNFL) thickness. GLP-1RA therapy was associated with reduced headache incidence (RR = 0.74, 95% CI: 0.54-1.00) and fewer monthly headache days (SMD = -0.77, 95% CI: -1.35 to -0.19). Changes in BMI were not significant (SMD = -0.06, 95% CI: -0.15 to 0.02).

CONCLUSIONS: GLP-1RAs show promise as adjunctive therapies for IIH, offering improvements in refractory IIH, papilledema, and headache burden. Further large-scale randomized controlled trials are needed to confirm these findings.

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Potential therapeutic role of GLP-1 receptor agonists in Idiopathic intracranial hypertension: a systematic review and meta-analysis

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