There is no FDA-approved drug for pediatric migraine in children aged younger than 12, so treatment is consensus-based rather than evidence-based.
Treatment with topiramate and amitriptyline did not significantly reduce headache frequency or headache-related disability in children and adolescents with migraine compared with placebo during the course of a 24-week study. In addition, the study drugs were associated with a higher rate of adverse events among patients assigned to them, according to the results of the CHAMP study published in The New England Journal of Medicine.
The trial was stopped early due to futility.
“During the trial, the FDA approved topiramate for the treatment of episodic migraine in adolescents 12 to 17 years of age,” wrote researchers led by Scott W. Powers, PhD, from the department of pediatrics at University of Cincinnati College of Medicine. “Although our trial included patients outside this age range and included those with either episodic or chronic migraine, the trial results suggest that prevention medication for pediatric migraine might be reexamined.”
There is currently no FDA-approved medication to treat pediatric migraine in children aged younger than 12. Because of this, current guidelines for the treatment of pediatric migraine are consensus-based rather than evidence-based.
The CHAMP trial was designed to test whether topiramate or amitriptyline reduced the frequency or severity of migraine in children and adolescents. Powers and colleagues enrolled 361 patients aged 8 to 17 and randomly assigned them 2:2:1 to amitriptyline 1 mg/kg per day, topiramate 2 mg/kg per day, or placebo; 328 were in the primary efficacy analysis. The primary outcome was a relative reduction of 50% or greater in the number of headache days compared with the 28-day baseline period.
A little over one-half of patients assigned to amitriptyline (52%) and topiramate (55%) achieved the primary outcome compared with 61% of patients assigned to placebo. There were no significant differences between the groups in headache-related disability, headache days, or the percentage of patients who completed the 24-week treatment period.
However, those patients assigned to study drugs did have a higher rate of adverse events. Patients assigned amitriptyline had higher rates of fatigue (30% vs. 14%) and dry mouth (25% vs. 12%), and those patients assigned topiramate had higher rates of paresthesia (31% vs. 8%) and weight loss (8% vs. 0%).
“Given the null outcome in this trial and the adverse events and serious adverse events reported in the amitriptyline and topiramate groups, the data do not show a favorable risk–benefit profile for the use of these therapies in pediatric migraine prevention, at least over the 24-week duration of the trial,” the researchers concluded.
Powers SW, et al. Trial of amitriptyline, topiramate, and placebo for pediatric migraine. N Engl J Med. Epub 2016 Oct 27.