GLP-1 Receptor Agonists Promising in Managing Idiopathic Intracranial Hypertension: JAMA Study

IIH, a disorder marked by increased pressure around the brain without an apparent cause, typically affects overweight women of reproductive age. The standard treatments—such as acetazolamide, topiramate, or lifestyle modifications—often have limited success and may not fully address the condition’s complex presentation. Given the known benefits of GLP-1 RAs in promoting weight loss and metabolic health, researchers led by Dr. Georgios S. Sioutas from the Department of Neurosurgery at Virginia Commonwealth University explored their therapeutic role in IIH.

Using real-world data from the TriNetX US Collaborative Network, the study analyzed health records from 67 healthcare organizations across the United States, covering the time frame from 2005 to 2024. Out of over 44,000 patients diagnosed with IIH, a matched cohort of 1,110 individuals was created, comparing 555 patients treated with GLP-1 RAs to 555 managed with conventional therapies.

The study reported the following findings:

• GLP-1 RA use was linked to a notable reduction in medication dependence, with a risk ratio (RR) of 0.53.
• Patients experienced fewer headaches (RR, 0.45) during the one-year follow-up.
• Visual disturbances and instances of blindness were less frequent in the GLP-1 RA group (RR, 0.60).
• A marked decrease in papilledema (optic nerve swelling) was observed, with an RR of 0.19.
• The need for procedural interventions was lower among patients receiving GLP-1 RA therapy (RR, 0.44).
• In this group, mortality rates were also reduced (RR, 0.36) over the follow-up period.
• These improvements occurred even though there was no significant difference in body mass index (BMI) between the GLP-1 RA group and the conventional treatment group at follow-up.
• Sensitivity analysis confirmed similar outcomes for patients regardless of whether their BMI was above or below 40.
• While bariatric surgery achieved more substantial weight loss, GLP-1 RA therapy was associated with more favorable overall clinical outcomes in patients with idiopathic intracranial hypertension.

The findings suggest that GLP-1 receptor agonists could represent a new direction in the treatment of IIH, particularly for patients who are not candidates for surgical intervention or who struggle with traditional medications.

The authors recommend that future prospective trials further investigate the role of GLP-1 RAs in IIH management. Given the burden of this condition and the limitations of current therapies, these results could pave the way for more effective, less invasive treatment strategies for patients living with IIH.

Reference:

Sioutas GS, Mualem W, Reavey-Cantwell J, Rivet DJ. GLP-1 Receptor Agonists in Idiopathic Intracranial Hypertension. JAMA Neurol. Published online July 14, 2025. doi:10.1001/jamaneurol.2025.2020