Effectiveness of onabotulinumtoxinA (BOTOX) in pediatric patients experiencing migraines: a randomized, double-blinded, placebo-controlled crossover study in the pediatric pain population

Introduction: OnabotulinumtoxinA (OBTA) is approved for treating chronic headaches and migraines in adults, but there is limited scientific literature on the outcomes in pediatric patients. The aim of this study was to determine if subjects treated with OBTA reported a statistically significant improvement in the primary features (frequency, intensity, duration and disability scoring) associated with migraines compared with placebo at follow-up visits.

Methods: After obtaining approval by the appropriate local (HS# 2016-3108) and federal institutions, the principal investigator enrolled candidates aged 8 to 17 years old diagnosed with chronic migraines (at least 6 months), and 15 or more headache days in a 4-week baseline period. This randomized control trial consisted of two phases: double-blind and open-label for the first two and last two sets of treatments, respectively. Subjects were randomly assigned to receive a treatment protocol-155 units at 31 injection sites-in 3-month intervals and follow-up visits every 6 weeks. Non-parametric testing (Wilcoxon signed-rank test) was performed using widely available open-source statistical software ('R').

Results: From February 2017 to November 2018, 17 subjects presented for a screening visit; 15 met eligibility criteria. Subjects that received OBTA reported a statistically significant decrease from the following baseline values compared with placebo 6-week post-treatment compared with placebo: frequency (20 (7 to 17) vs 28 (23 to 28); p=0.038), intensity (5 (3 to 7) vs 7 (5 to 9); p=0.047), and PedMIDAS (Pediatric Migraine Disability Score) (3 (2 to 4) vs 4 (4 to 4); p=0.047). There was no statistically significant difference in the duration (10 (2 to 24) vs 24 (4 to 24); p=0.148) of migraines between the two groups.

Discussion: OnabotulinumtoxinA showed a statistically significant decrease in frequency and intensity of migraines compared with placebo. No adverse effects or serious adverse events related to the use of OBTA were reported. In the future, we aim to evaluate the specific nature of migraines, for example, quality/location of pain presented during an initial consult to predict the likelihood of OBTA being a truly effective modality of pain management for pediatric migraineurs.

Trial registration number: NCT03055767.