Key takeaways:

• Botox injections are FDA-approved to treat headaches in adults but are not approved in adolescents due to a lack of data.
• A third of patients saw a more than 50% reduction in headache days with two treatments.

Adolescents and young adults with chronic migraines saw significant reductions in daily headaches and in headache disability scores after treatments of onabotulinumtoxin A, according to data published in Pediatric Neurology.

The study is the first prospective study to show the efficacy of onabotulinumtoxin A (Botox, AbbVie) injections in reducing headache days in the pediatric population, specifically those with daily chronic migraines, said Jeffrey Strelzik, MD, a neurologist with Children’s National Hospital.

Data derived from Vilardo L. et al. Pediatr Neurol. 2026:doi:10.1016/j.pediatrneurol.2026.07.009.

“This is an option for pediatric patients that works, or seems to lead to improvement, and should be considered by anybody who is looking for good options when they feel like they are running into a refractory situation,” Strelzik told Healio.

Although Botox injections have been FDA-approved for adult migraines, they have not been approved for pediatric migraines due to a lack of consistent data in children with chronic migraines, authors wrote.

Chronic migraine, defined as experiencing headaches more than 15 days per month for more than 3 months, can be a debilitating challenge for children, Strelzik said.

Children who experience “more than 10 migraine days per month have a higher prevalence of learning disabilities and attention deficit hyperactivity disorder,” authors wrote, citing research published in 2020 in Current Pain and Headache Reports.

“In real life, these are kids who have had to drop sports, who have had to take semesters off from school,” Strelzik said. “These are kids who are missing 2 to 3 days of school a week.”

The method

Strelzik and colleagues examined 45 patients with chronic migraine between the ages of 12 and 22 years (median age, 17 years; 80% women) who had already failed at least two preventative medications.

A subgroup analysis was performed on 27 patients with daily chronic migraines. This subgroup has been historically resistant to treatment and is often excluded from studies, Strelzik said.

“There are very few studies that look at these kids with new daily persistent headache, or headaches that are happening daily, because the very high likelihood is that they will not see a response to whatever their treatment is,” he said.

All patients in the study were treated with Botox every 3 months with a total dose of 155-200 units per treatment.

Results were compiled through a series of surveys completed before and after Botox treatments that assessed headache frequency, pain levels, abortive use and quality of life. The study period ran from March 2020 through April 2024.

Roughly 27% of patients saw a more than 50% reduction in headache days after the first treatment (P < .0005). Of the 24 patients to receive a second treatment, 33% of them saw a more than 50% reduction in headache days (P = .004).

Median baseline headache frequency was 28 days per the last 28 days. After one treatment, the median frequency fell to 20 days (P = .004), and it fell to 12 days after two treatments (P = .007).

The significant changes after one treatment were notable, Strelzik said. In studies into Botox’s effects on adults with migraines, most patients did not improve after the first dose, he said.

“Maybe that’s a difference between the pediatric population and the adult population, or maybe there’s another factor that’s contributing there, but I think it’s a meaningful thing to explore further that I think most people haven’t considered,” Strelzik said.

Among the 27 patients with daily chronic migraine, 22% of them had a more than 50% reduction in headache days after one treatment (P = .016). After the first treatment, mean headache days fell to 22 days (P = .016), and it continued to fall to 21 days after the second treatment (P = .009).

These findings are particularly notable because of the refractory and debilitating headaches experienced by this subgroup, Strelzik said.

“To have something that a quarter of the time makes a difference is actually a giant, giant leap forward for this cohort of patients who already come to us at Children’s National from far around our region,” Strelzik said.

Strelzik and colleagues measured headache disability using the Headache Impact Test, also called HIT-6.

The mean HIT-6 score at baseline was 62, which decreased to 58 after the first treatment (P = .0017) and to 57 after the second treatment (P = .004). The mean remained at 57 after the third treatment (P = .0026) and fell to 55 after the fourth treatment (P = .005).

The baseline mean HIT-6 scores for the daily chronic migraine subgroup was 64. The difference was not significant after one treatment, but the mean scores after the second and third treatments were both significant, with scores of 57 (P = .001) and 56 (P < .005), respectively.

Mean headache disability decreased for the entire cohort after four treatments.

One study limitation was the lack of a placebo group, which Strelzik said can be difficult to navigate for pediatric studies using Botox injections.

“To do that in kids where we know that they are a little bit more susceptible to pain ... and not actually deliver a drug, there are some moral and ethical questions there that are harder to address,” he said. “There have been some more randomized trials in the past for Botox, so I’m not saying it’s not doable, but it would be a very, very different protocol.”

Moving forward, Strelzik said a comparison group is important to better characterize the placebo effect. He also said studies with larger cohorts will provide useful data.

In the meantime, Strelzik said the findings from him and his colleagues should make physicians consider implementing Botox as an option for patients who did not see results from other medications.

“If they don’t know how to do Botox, they should consider learning,” Strelzik told Healio. “And if they are already doing Botox, then they should maybe push some of these patients toward it a little earlier now that we have some data.”

For more information:

Jeffrey Strelzik, MD, can be reached at neurology@healio.com.