Evaluation of the added value of botulinum toxin treatment and intensive physiotherapy to improve body function and structure in children with spastic cerebral palsy

INTRODUCTION: Botulinum neurotoxin injections (BoNT-A) followed by a period of intensive physiotherapy (iPT) and, if necessary, casting or ankle-foot orthoses, form a widely-used treatment combination for ambulatory children with spastic cerebral palsy (CP). The added value of BoNT-A for improving body function and structure has not been explicitly studied, however. PATIENTS AND METHOD: A comparative effectiveness multicentre trial was performed. Children with spastic CP, aged 4- 12 years, GMFCS levels I-III, were either randomised to BoNT-A+iPT or only-iPT, or allocated to their preferred treatment group. Outcomes for effectiveness were related to body function and structure, and were assessed at baseline, 12 and 24 weeks. We performed intention-to-treat analysis with linear mixed models. RESULTS: 65 children participated (40% randomized): 41 children received BoNT-A+iPT and 24 received only-iPT. Participants were equally distributed over GMFCS levels, their mean age was 7.3 years. We did not find any statistically significant differences in treatment effects between groups, except for gait-analysis-measured knee angle during midstance while wearing footwear at 12 weeks, which was in favour of only-iPT (4.8 degrees difference between groups, 95% CI 0.2 to 9.2, p=0.036), and gait-analysis-measured maximum knee angle during swing while barefoot at 24 weeks, which was in favour of BoNT-A+iPT: (5.4 degrees difference, 95% CI -0.4 to 10.5, p=0.035). CONCLUSION: At the group level, BoNT-A injections did not add to clinical effectiveness in combined BoNT-A+iPT treatment. This suggests that prescription and use of BoNT-A for improvement of outcomes concerning impaired functioning has to be critically reconsidered in this CP age and severity subgroup.