OP33 – 2365: Treatment with botulinum toxin of children with cerebral palsy has no added therapeutical value or cost-effectiveness for gross motor function, everyday physical activity levels or quality of life when combined with intensive functional physiotherapy

Abstract

Objective Since 20 years, children with spastic CP are increasingly treated with botulinum toxin typeA (BtA) followed by intensive physiotherapy (iPT). The added value of BtA remains unclear. We therefore compared (cost-)effectiveness of BtA+iPT versus only iPT treatment in a (partly) randomized controlled trial. Methods Sixty-five children aged 4–12 years participated, 41 received BtA+iPT and 24 only iPT. Measurements were performed after a 12-week iPT period (primary endpoint) and 12 weeks thereafter (24week follow-up). Primary outcome measures were: actual everyday physical activity levels measured with ambulatory monitoring devices (Actigraph-AG, 7day monitoring), (health related) quality of life measured with several questionnaires (Health Utility Index, Child Health Questionnaire, DISABKIDS-questionnaires), and gross motor function measured with the GMFM (item set version). Economic evaluation was performed according to Dutch guidelines taking a societal perspective. Results No statistically significant evidence was found for added value of BtA injections in BtA+iPT treatment for the primary outcomes. At primary endpoint, we found trends towards an intervention effect in favor of only iPT for improving gross motor function (GMFM; p=0.095, ES=1.12, 95%CI[-0.20:2.44]) and decreasing the % sedentary behavior during everyday physical activity (AG%sed; p=0.087, ES=–2.59, 95%CI[-5.58:0.39]). At 24week follow-up, we found a trend – also in favor of iPT – towards improving quality of life (DISABKIDS-smileys-proxy-version; p=0.066, ES=5.9, 95%CI[-0.40:12.20]). We found one trend in favor of BtA for improving everyday physical activity counts per minute (AGcpm; p=0.064, ES=91, 95%CI[-6:188]). Average treatment costs (including plaster and AFO) were significantly higher in BtA+iPT than in iPT (8.963 euro versus 6.182 euro, p=0.001). Conclusion We conclude that adding BtA to iPT in combined BtA+iPT treatment does not improve effectiveness (at the group level) and that adding BtA is also not cost-effective when treatment is aimed at improving gross motor function, everyday physical activity levels or quality of life.