OBJECTIVE: This study evaluated the efficacy of glucagon-like peptide-1 receptor agonists (GLP-1-RAs) in idiopathic intracranial hypertension (IIH), focusing on their effects on clinical outcomes, management escalation, and mortality.
METHODS: The authors conducted a cohort study using the TriNetX Research Network, comparing IIH patients treated with GLP-1-RAs to untreated patients, employing propensity score matching. Clinical outcomes, including headaches, visual and cognitive deficits, acetazolamide use, surgery, and mortality, were assessed at 6-month and 1-year follow-up (FU).
RESULTS: At 6-month FU, 5750 patients in the GLP-1-RA cohort were matched to 5750 in the control group. At 1-year FU, 4968 patients in the GLP-1-RA cohort were matched to 4968 in the control group. The GLP-1-RA group demonstrated a significant reduction in BMI (p < 0.001) at 6 months, with a standardized mean difference of 1.083 kg/m2, which increased to 1.635 kg/m2 at 1 year. The control group showed a smaller reduction (p = 0.006), with a standardized mean difference of 0.695 kg/m2 at 6 months and 0.758 kg/m2 at 1 year. Furthermore, GLP-1-RA users had significantly lower odds of new-onset headache (OR 0.660, 95% CI 0.543-0.799, p < 0.001), visual deficits (OR 0.423, 95% CI 0.324-0.546, p < 0.001), cognitive deficits (OR 0.368, 95% CI 0.246-0.539, p < 0.001), and acetazolamide use (OR 0.295, 95% CI 0.249-0.348, p < 0.001) at 6 months. These trends persisted at 1 year for visual deficits (OR 0.606, 95% CI 0.489-0.747, p < 0.001), cognitive deficits (OR 0.590, 95% CI 0.432-0.801, p = 0.006), and acetazolamide use (OR 0.374, 95% CI 0.320-0.437, p < 0.001). Shunt placement for GLP-1-RA users also showed significantly lower risk at 1 year (OR 0.375, 95% CI 0.171-0.753, p = 0.047). Mortality rates were lower in the GLP-1-RA group at both 6 months (OR 0.060, 95% CI 0.031-0.106, p < 0.001) and 1 year (OR 0.115, 95% CI 0.070-0.179, p < 0.001). Kaplan-Meier survival curves confirmed these findings, additionally showing cumulative significance for headache reduction (p = 0.008).
CONCLUSIONS: GLP-1-RAs may provide clinical benefits for patients with IIH, improving outcomes and reducing the need for invasive interventions. Future randomized, prospective studies are warranted to confirm these findings and optimize treatment strategies.
