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Topiramate Has Not Proven Efficacious In Pediatric Migraine Prophylaxis


  • In children with frequent migraines requiring prophylaxis, is topiramate effective for decreasing migraine frequency?

Clinical Bottom Lines

  1. Topiramate has been show to be effective in adults for migraine prophylaxis, but this study did not show efficacy of topiramate in the pediatric population, compared to placebo.
  2. For the primary outcome (reduction in number of migraine days per month compared to baseline phase), there was no statistically significant difference between those receiving topiramate versus placebo.
  3. There was a statistically significant reduction in number of migraine days per month for topiramate-treated patients during the last 28 days of treatment.

Summary of Key Evidence

1. Industry-sponsored, randomized, double-blind, placebo-controlled study of 162 children at 17 non-academic clinics.
2. Patients were 6-15 years old with 3-10 migraines/month for 3 months before screening and during 4 week baseline phase.
3. Patients were excluded for cluster headache, chronic migraine, >15 migraines in the 4 week baseline phase, overused analgesics/abortives, had previously failed topiramate or any 2 prophylactic medications, or had a history of nephrolithiasis.
4. Headache diaries were used to track frequency, severity, duration, associated symptoms and use of analgesics/abortives.
5. Symptoms were assessed during a 4 week baseline phase, followed by an 8 week titration phase (up to 2-3mg/kg/d divided BID, or maximum dose of 200mg/day), and a 12 week maintenance phase. Average maintenance dose was 2mg/kg/d.
6. Analyses were performed on the intent-to-treat population (n=157) and the per-protocol population (n=126, patients who completed study without major protocol violations).
7. For the primary endpoint, reduction in number of migraine days per month during the titration and maintenance phases (for ITT group), there was no significant difference between groups (2.6 days/month for topiramate vs. 2 days/month for placebo; p=0.061). However, there was a significant difference for the per-protocol population (2.8 days/month for topiramate, 2.2 days/month for placebo; p=0.033).
8. During the last 28 days of treatment for the ITT population, there was a significant difference in the reduction of migraine days/month (3.1 days/month for topiramate, 2.4 days/month for placebo; p=0.023).

Additional Comments

  • 1. A smaller study conducted in India did show a statistically significant decrease in monthly migraine frequency with topiramate versus placebo. However, the children in this study had more migraine days/month at baseline compared to the above study.2
    2. Overall, larger studies are required to further assess topiramate efficacy for pediatric migraine prophylaxis, and greater attention to functional outcomes is warranted.


  1. Winner P, et al. Topiramate for migraine prevention in children: a randomized, double-blind, placebo-controlled trial. Headache 2005; 45:1304-1312.
  2. Lakshmi CVS, et al. Topiramate in the prophylaxis of pediatric migraine; a double-blind, placebo-controlled trial. J Child Neurol. 2007; 22:829-835.

CAT Author: Kristin Hyman, MD

CAT Appraisers: James Gurney, PhD

Date appraised: April 15, 2009

Last updated June 11, 2009
Department of Pediatrics and Communicable Diseases
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