Potential Pediatric Use of New Migraine Therapies
Over the past 10 years multiple new treatments have been approved for use in adults. However, there has been little improvement in treatments specifically indicated for treatment of migraine in children, and very little data on the effects of these medications in children are available. At the Virtual Scottsdale Headache Symposium on November 21, 2020, Chaouki Khoury, MD reviewed the data that are available for treatments as diverse as botunlinumtoxinA to lasmiditan.
A randomized controlled trial of botulinumtoxinA injection vs placebo saline injections failed to show a statistically significant difference between the treatments after only 1 round of injections but did establish safety. Unfortunately, this study did not examine results after 2 rounds of injections, known from adult studies typically to be needed before effects of treatment are seen. There have been 3 retrospective studies of 10 to 30 children with chronic migraine that used the standardized regimen of 31 injections of 155 U with multiple rounds of injection. In all 3 studies, there was a statistically significant reduction in migraine frequency, such that it became episodic rather than chronic. Reductions in headache intensity and duration were also observed.
Several neuromodulation devices are available and FDA-approved for adults; an advantage is the nonpharmacologic nature of these treatments, but this is offset by the high cost of most of the devices. For children, only the transcranial magnetic stimulator is approved after a study in 12 children. A newer device, the noninvasive vagal nerve stimulator has been studied in children and 22 of 47 attacks (46%) did not require subsequent pharmacologic treatment; this is not approved for children and the cost remains prohibitive.
Calcitonin gene-related peptide (CGRP) inhibitors are not yet approved for use in children, although increased levels of have been observed in children with migraine during attacks. Ongoing clinical trials are recruiting pediatric participants age 6 to 17 years to study whether these new drugs can be safe, effective treatments in children. No data are available for the CGRP inhibitor acute treatments of migraine in children.
For use of any of these migraine treatments in children, insurance companies are likely to at first deny coverage. This can be better appealed by documentation of 2 or more medication failures for preventive treatments. For abortive treatments, the more documented times medication has failed to effectively treat a child's migraine, the more likely approval will be granted. Sometimes peer-to-peer review is required, and even then a "plan exclusion" may be invoked to deny coverage.
Dr. Khoury concluded by saying that many of the new migraine treatments are more specific to migraine pathophysiology with safer tolerablity profiles and should not be withheld from children experiencing the pain and discomfort of migraine.