Effect of upper limb botulinum toxin-A therapy on health-related quality of life in children with hemiplegic cerebral palsy

Aim:   Currently, the use of upper limb botulinum toxin-A (UL BTX-A) is based on evidence of functional efficacy without supporting evidence of positive change in health-related quality of life (HRQOL). While function may improve, this cannot be directly correlated with an improvement in HRQOL. Most paediatric studies use caregiver/parent proxy reports. The inclusion of child self-reports is increasing as poor correlation with proxy reports is being demonstrated. This paper aims to study the effect of UL BTX-A therapy on HRQOL in children with hemiplegic cerebral palsy (CP).
Method:   Design: Pilot prospective randomised trial. Participants: 22 children with hemiplegic CP aged 7 years 0 month?13 years 11 months (12 treatment, 10 control). Treatment: One series BTX-A injections into UL. HRQOL assessed at baseline, and 1, 3 and 6 months post-injection by completion of Pediatric Quality of Life (PedsQL) 4.0 Generic Core Scales and PedsQL 3.0 CP Module. Outcome: 1. Change in PedsQL scores. 2. Concordance between child self-report and parent proxy-report scores.
Results:   No statistically significant difference between treatment and control groups was observed for any domain of HRQOL. Intraclass concordance was good for the PedsQL CP Module Daily Activities, and Speech and Communication scores ( P  = 0.0005).
Conclusion:   This pilot work adds to the emerging evidence that UL BTX-A therapy has no statistically significant effect on the HRQOL of children with hemiplegic CP. With the increasing use of this therapy in children with CP, further research across the broader CP population is needed to identify whether this therapy is indicated in other target populations. Both child and parent proxy reports should be collected when assessing HRQOL in this population.