AUTHOR=Jacobson Dan , Löwing Kristina , Kullander Kjell , Rydh Britt-Marie , Tedroff Kristina
TITLE=A First Clinical Trial on Botulinum Toxin-A for Chronic Muscle-Related Pain in Cerebral Palsy
JOURNAL=Frontiers in Neurology
VOLUME=Volume 12 - 2021
YEAR=2021
URL=https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2021.696218
DOI=10.3389/fneur.2021.696218
ISSN=1664-2295
ABSTRACT=Objective: To test if botulinum toxin-A (BoNT-A) is effective in reducing chronic muscle-related pain in adults with spastic cerebral palsy (CP), as compared to placebo.
Design: A single-center, double-blind, parallel, randomized placebo-controlled trial. The design included an interim analysis to allow for confirmatory analysis, as well as pilot study outcomes.
Setting: Tertiary university hospital.
Participants: Adults with spastic CP and chronic pain associated with spastic muscle(s).
Intervention: Treatment was one session of electromyographically guided intramuscular injections of either BoNT-A or placebo normosaline.
Main Study Outcomes: The primary outcome was the proportion who achieved a reduction of pain intensity of two or more steps on the Numerical Rating Scale 6 weeks after treatment.
Results: Fifty individuals were screened for eligibility, of whom 16 were included (10 female, 6 male, mean age = 32 years, SD = 13.3 years). The randomization yielded eight participants per treatment arm, and all completed the study as randomized. The study was stopped at the interim analysis due to a low probability, under a preset threshold, of a positive primary outcome. Four individuals were treatment responders in the BoNT-A group for the primary outcome compared to five responders in the placebo group (p = 1.000). Adverse events were mild to moderate. In exploratory analysis, the BoNT-A group had a trend of continuing reduction of pain at the last follow-up, after the primary endpoint.
Conclusions: This study did not find evidence that BoNT-A was superior to placebo at the desired effect size (number needed to treat of 2.5) at 6 weeks after treatment.
Trial registration:ClinicalTrials.gov: NCT02434549