We present the first known pediatric cases of idiopathic intracranial hypertension (IIH) treated successfully with glucagon-like peptide-1 receptor agonists (GLP-1 RAs).

IIH causes significant morbidity including disabling headaches and permanent vision loss in up to 25% of cases. The incidence and prevalence of IIH is rising, likely due to the increasing prevalence of obesity worldwide. GLP-1 receptor agonists (GLP-1 RAs) are commonly used in the treatment of diabetes and obesity. Weight loss is the mainstay of treatment for IIH, and recent evidence has shown that GLP-1 RAs have been effective for headache management in non-pediatric populations.

Patients (n=2) had a mean age of 16, mean pre-treatment BMI of 40, and headaches. They presented to our tertiary care children’s hospital after they were found to have bilateral papilledema on ophthalmologic examination. Patient 1 had initial lumbar puncture (LP) opening pressure of 34 cm H₂O, and he was treated with therapeutic LPs and acetazolamide (ACZ). There was consideration of shunt placement due to persistent elevated intracranial pressure (ICP). He eventually started exenatide and had almost complete resolution of headaches at his 5 month follow up visit, despite minimal weight changes. Patient 2 had LP opening pressure maximum of 40 cm H₂O. She continued to have papilledema and headaches despite therapy with ACZ, and failed topiramate due to nephrolithiasis. She started tirzepatide and had mild improvement in headaches after 3 months. Mean post-treatment BMI was 35.5.