Prescription medicine used to treat migraines in children are no more effective than placebo sugar pills, says a study in The New England Journal of Medicine.
The study showed no significant differences among amitriptyline, topiramate and placebo in reducing headache days or related disability.
“The study was intended to demonstrate which of the commonly used preventive medications in migraine was the most effective. What we found is that we could prevent these headaches with either a medication or a placebo,” says Andrew Hershey, MD, PhD, co-director of the Cincinnati Children’s Headache Center and senior author of the study.
“This study suggests that a multi-disciplinary approach and the expectation of response is the most important, not necessarily the prescription provided,” he added.
The 24-week clinical trial included 328 eligible pediatric patients. The trial used a clinically meaningful endpoint of a 50 percent or greater reduction in headache days from the 28 days prior to randomization to the final four weeks of the 24-week study.
The surprise finding was that 61 percent of kids taking a placebo saw the days they had a headache reduced by 50 percent or more.
For the two medication groups, 52 percent of those taking amitriptyline and 55 percent of those taking topiramate had this level of reduced headache days.
The rates among the three groups were not statistically different.
Compared to placebo, those on the two active drugs had a significantly higher rate of side effects, including fatigue, dry mouth and, in three cases, mood alteration. Thirty-one percent of those on topiramate had paresthesia – a “pins and needles” tingling in the hands, arms, legs or feet.
These results create a bit of a conundrum given that it is not ethical to prescribe placebo without a patient’s knowledge, and it may be the expectation of reduced migraine may override the actual effects of the medicines.
“Our national team was hoping to develop evidence to drive the choice by medical providers of the first line prevention medication for helping youth with migraine, but the data showed otherwise,” said Scott Powers, PhD, pediatric psychologist, co-director of the headache center at Cincinnati Children’s, and first author of the paper.
“We see this as an important opportunity for health care providers, scientists, children, and families because our findings suggest a paradigm shift. First line prevention treatment will involve a multidisciplinary team approach and focus on non-pharmacological aspects of care. The good news is we can help children with migraines get better,” he added.
Powers says the study also underscores the importance of conducting more research with a developmental focus on children and young adults to allow innovations that can be applied directly to pediatric chronic illnesses.
“The interpretation of these results is very challenging. In most situations, trials that fail to show benefit of an intervention do so because study participants do not improve. That was not the situation here. A majority of all study participants improved, regardless of their assigned treatment group,” says Chris Coffey, PhD, director of the CTSDMC and professor of biostatistics in the University of Iowa’s College of Public Health, and lead statistician for the study.
“Further research is needed to better understand the results and to determine what future strategies might optimize the treatment of headaches in these childhood and adolescent populations,” he added.