Fabienne Schasfoort, Annet Dallmeijer, Robert Pangalila, Coriene Catsman, Henk Stam, Jules Becher, Ewout Steyerberg, Suzanne Polinder, Johannes Bussmann
Rehabilitation Medicine, Erasmus MC, NL-3000 CA Rotterdam, The Netherlands. E-mail: f.schasfoort@erasmusmc.nl
DOI: 10.2340/16501977-2267

Objective: Despite the widespread use of botulinum toxin in ambulatory children with spastic cerebral palsy, its value prior to intensive physiotherapy with adjunctive casting/orthoses remains unclear.
Design: A pragmatically designed, multi-centre trial, comparing the effectiveness of botulinum toxin + intensive physiotherapy with intensive physiotherapy alone, including economic evaluation.
Subjects/patients: Children with spastic cerebral palsy, age range 4–12 years, cerebral palsy-severity Gross Motor Function Classification System levels I–III, received either botulinum toxin type A + intensive physiotherapy or intensive physiotherapy alone and, if necessary, ankle-foot orthoses and/or casting.
Methods: Primary outcomes were gross motor func-tion, physical activity levels, and health-related quality-of-life, assessed at baseline, 12 (primary end-point) and 24 weeks (follow-up). Economic outcomes included healthcare and patient costs. Intention-to-treat analyses were performed with linear mixed models.
Results: There were 65 participants (37 males), with a mean age of 7. 3 years (standard deviation 2. 3 years), equally distributed across Gross Motor Function Classification System levels. Forty-one children received botulinum toxin type A plus intensive physio-therapy and 24 received intensive physiotherapy treatment only. At primary end-point, one statistically significant difference was found in favour of intensive physiotherapy alone: objectively measured percentage of sedentary behaviour (–3. 42, 95% confidence interval 0. 20–6. 64, p=0. 038). Treatment costs were significantly higher for botulinum toxin type A plus intensive physiotherapy (8,963 vs 6,182 euro, p=0. 001). No statistically significant differences were found between groups at follow-up.
Conclusion: The addition of botulinum toxin type A to intensive physiotherapy did not improve the effectiveness of rehabilitation for ambulatory children with spastic cerebral palsy and was also not cost-effective. Thus botulinum toxin is not recommended for use in improving gross motor function, activity levels or health-related quality-of-life in this cerebral palsy age- and severity-subgroup.

For children with spastic cerebral palsy, botulinum toxin injections are often part of a combined treatment package, that usually also includes a physiotherapy period and additional casting or orthoses. It is known that such a combined package is effective, but up to now the botulinum toxin contribution to the total treatment effect is unclear. Our study is the first to specifically study the added value of the botulinum toxin injections for improving gross motor function, physical activity levels and/or quality of life in spastic cerebral palsy. In a group of 65 children aged 4 to 12 years, we found no differences in effect between the group that received the package with injections and the group that did not. Therefore, prescription and use of botulinum toxin for these treatment goals needs critical reconsideration.

Botulinum toxin type A for spastic cerebral palsy: Is it time to change praxis?
Intramuscular botulinum toxin prior to comprehensive rehabilitation has no added value for improving motor impairments, gait kinematics and goal attainment in walking children with spastic cerebral palsy