Abstract 8: Functional and societal limitation and disability outcomes in a randomized control trial of botulinum toxin type a for children with spastic diplegic cerebral palsy.

Abstract

Objectives: To document the functional and societal limitation and disability level effects (National Center for Medical Rehabilitation Research framework) of gastrocnemius botulinum toxin type A (BTX) injections in children with spastic diplegic cerebral palsy (CP). Design: Randomized double-masked placebo controlled trial. Setting: Tertiary care children’s hospital. Participants: 33 children with spastic diplegic CP, (mean age, 5.5y; range, 3.0–11.9y), of whom 19 were boys (subjects’ Gross Motor Function Classification System levels: level I=12; level II=15; level III=6). Intervention: All participants were randomized to receive either 12U/kg BTX or placebo saline injections. Main Outcome Measures: Functional limitation and disability: Gross Motor Function Measure (GMFM-88, GMFM-66), energy cost index (ECI), and Canadian Occupational Performance Measure (COPM) performance scores were collected at selected weeks postinjection. Societal limitation: COPM satisfaction scores and Goal Attainment Scaling (GAS) were obtained at 12 and 24 weeks. Results: GMFM-88 total score ( P=.00) and the GMFM-66 ( P=.03) significantly increased for the treatment group compared with the placebo group at 24 weeks. No significant group differences were found for the ECI at any follow-up point. The COPM performance scores were significantly greater for the treatment group at 12 weeks ( P=.04) and approached a significant difference at 24 weeks ( P=.06). COPM satisfaction scores and GAS did not differ significantly by group at any follow-up point. Conclusions: The GMFM-88 and -66 scores and COPM performance scores demonstrated a significant difference at 12 weeks, with a diminishing effect noted at 24 weeks. In contrast, satisfaction with performance as measured by the COPM satisfaction scores and performance and importance of goals set as measured by the GAS did not differ significantly between treatment groups. These data documented consistent changes on the functional limitation and disability levels up to 6 months posttreatment, but not on the societal outcome level.