Children’s National study finds Botox cuts pediatric chronic migraine headaches

A prospective study led by researchers at Children’s National found that onabotulinumtoxinA (ONA), commonly known as Botox, reduced headache frequency and headache-related disability in children and young adults with chronic migraine, adding important evidence to support its use in a pediatric population where treatment options remain limited.

The study, published in Pediatric Neurology, followed 45 patients ages 12 to 22 with chronic migraine who received ONA injections using the Phase II Research Evaluating Migraine Prophylaxis Therapy (PREEMPT-2) protocol at the Children’s National Headache Program.

While ONA is FDA-approved as a preventive treatment for adults with chronic migraine, it has not been approved for pediatric migraine because of limited and inconsistent data in children and adolescents. That lack of evidence has also created challenges for insurance coverage, limiting access for many families. “Our study helps address an important evidence gap,” said Jeffrey Strelzik, MD, director of headache injection at Children’s National and co-senior author of the study. “By demonstrating reductions in both headache frequency and disability, these findings contribute to the growing body of research supporting the use of onabotulinumtoxinA in appropriately selected pediatric patients.”

Key findings:

• 27% of patients experienced a greater than 50% reduction in headache days after one treatment.
• 33% of patients who received two treatments achieved a greater than 50% reduction in headache days.
• 22% of patients with daily chronic migraine experienced a greater than 50% reduction in headache days after one treatment.
• Headache-related disability significantly improved following treatment, suggesting better daily functioning and quality of life.

In addition to reducing headache frequency, ONA significantly lowered headache-related disability, as measured by the Headache Impact Test (HIT-6), suggesting meaningful improvements in patients’ daily functioning and quality of life.

“Chronic migraine can have a profound impact on a child’s education, social development and overall well-being,” said Dr. Strelzik. “Our findings suggest that onabotulinumtoxinA may be an effective treatment option for pediatric patients with chronic migraine, including some who experience headaches every day.”

Children’s National is among the few centers in the region with a dedicated Headache Injection Program for children and adolescents with chronic migraine. As part of the Headache Program, it offers a multifaceted approach that combines lifestyle changes, behavioral strategies, devices and procedures such as Botox injections to improve headache-related quality of life. The program performs more than 200 ONA procedures each year, offers peripheral nerve blocks and gives patients access to emerging migraine therapies through clinical trials, including studies of cGRP-targeted treatments.

The researchers say additional studies are needed to further define which patients are most likely to benefit from treatment and to continue building the evidence needed to improve access for children with chronic migraine.