The evidence-based approach to guide clinical practice has gained great importance in the medical field. High-quality evidence is of paramount importance to inform clinical decision-making and optimize patient outcomes. The generation of high-quality evidence relies on sound methodology and study design to facilitate appropriate interpretation and subsequent application of the clinical findings. The purpose of this review is to objectively critique the methodological design and clinical findings of 2 pediatric orthopaedic studies on children with cerebral palsy (CP) to assess their potential to impact clinical practice.Methods:
This is the second in a series of evidence-based reviews in pediatric orthopaedics. The pediatric orthopaedic literature was reviewed for randomized controlled trials (RCTs) published in 2015 and 2016. One RCT was selected from the journal Pediatrics, and one RCT was selected from the journal Developmental Medicine and Child Neurology, both investigating the use of botulinum toxin for the treatment of spastic equinus in children with CP. These RCTs were subjected to in-depth methodological review by orthopaedic surgeons with advanced research degrees and a PhD researcher. Two clinical experts then reviewed the articles to rate the clinical impact or value of each study. Methodological and clinical reviews were compiled, and a final recommendation on impact to change clinical practice was made based on both review components at the consensus of the panel.Results:
The first study reviewed investigated the single-dose efficacy and safety of botulinum neurotoxin type-A (1BoNT-A) in children with CP-associated spasticity and equinus foot deformity. The reviewers deemed the placebo-controlled study to be of sound design, and conclusions appropriate for the methodology used and clinical findings. Although findings suggest 1BoNT-A may provide benefit in treating equinus foot deformity, the optimal dose for achieving maximal impact on functional improvement remains undetermined. The second study reviewed investigated the impact of multiple dosing schedules on spastic equinus in CP. Although of relatively sound design, this study was limited by a small sample size and lack of justification for the chosen effect size. Without further study, no recommendation to change clinical practice could be made.Conclusions:
Both RCTs reviewed were superiority studies, the first demonstrating efficacy of botulinum toxin over placebo, the second showing no significant difference in 4- and 12-monthly botulinum toxin injections.Significance:
Despite a positive result demonstrating an effect of botulinum toxin treatment on spastic equinus in CP, the long-term functional impact and optimal dose remains to be determined. In addition, the negative result in the second study demonstrates the need for a noninferiority trial design to appropriately demonstrate no difference between 2 treatment options.