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


METHODS



METHODS
Pilot prospective randomised trial.


METHODS
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.


RESULTS
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).


CONCLUSIONS
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.
Telethon Institute for Child Health Research, University of Western Australia, Perth, Western Australia, Australia. toni_redman@health.qld.gov.au
Toni A
TA
Redman
Judith C
JC
Finn
Alexandra P
AP
Bremner
Jane
J
Valentine
D000293:Adolescent; D019274:Botulinum Toxins, Type A / Q000627:therapeutic use*; D002547:Cerebral Palsy / Q000188:drug therapy* / Q000503:physiopathology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006429:Hemiplegia / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009465:Neuromuscular Agents / Q000627:therapeutic use*; D010446:Peptide Fragments / Q000627:therapeutic use*; D011788:Quality of Life; D034941:Upper Extremity; D014904:Western Australia
D009465:Neuromuscular Agents; D010446:Peptide Fragments; C505468:botulinum neurotoxin A (870-1295); D019274:Botulinum Toxins, Type A