Botulinum toxin A for nonambulatory children with cerebral palsy: a double blind randomized controlled trial

Copeland, Lisa, Edwards, Priya, Thorley, Megan, Donaghey, Samantha, Gascoigne-Pees, Laura, Kentish, Megan, Lindsley, Jayne, McLennan, Kim, Sakzewski, Leanne and Boyd, Roslyn N. (2014) Botulinum toxin A for nonambulatory children with cerebral palsy: a double blind randomized controlled trial. Journal of Pediatrics, 165 1: 1-11. doi:10.1016/j.jpeds.2014.01.050

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Author Copeland, Lisa
Edwards, Priya
Thorley, Megan
Donaghey, Samantha
Gascoigne-Pees, Laura
Kentish, Megan
Lindsley, Jayne
McLennan, Kim
Sakzewski, Leanne
Boyd, Roslyn N.
Title Botulinum toxin A for nonambulatory children with cerebral palsy: a double blind randomized controlled trial
Journal name Journal of Pediatrics   Check publisher's open access policy
ISSN 0022-3476
Publication date 2014-03-14
Year available 2014
Sub-type Article (original research)
DOI 10.1016/j.jpeds.2014.01.050
Open Access Status
Volume 165
Issue 1
Start page 1
End page 11
Total pages 11
Place of publication Philadelphia, PA, United States
Publisher Mosby
Language eng
Formatted abstract
Objectives To examine the efficacy and safety of intramuscular botulinum toxin A (BoNT-A) to reduce spasticity and improve comfort and ease of care in nonambulant children with cerebral palsy (CP).

Study design Nonambulant children with CP (n = 41; Gross Motor Function Classification System level IV = 3, level V = 38; mean age 7.1 years, range 2.3-16 years, 66% male) were randomly allocated to receive either intramuscular BoNT-A injections (n = 23) or sham procedure (n = 18) combined with therapy. The analysis used generalized estimating equations with primary outcome the Canadian Occupational Performance Measure (COPM) at 4 weeks postintervention and retention of effects at 16 weeks. Adverse events (AE) were collected at 2, 4, and 16 weeks by a physician masked to group allocation.

Results There were significant between group differences favoring the BoNT-A-treated group on COPM performance at 4 weeks (estimated mean difference 2.2, 95% CI 0.8, 3.5; P = .002) and for COPM satisfaction (estimated mean difference 2.2, 95% CI 0.5, 3.9; P = .01). These effects were retained at 16 weeks for COPM satisfaction (estimated mean difference 1.8, 95% CI 0.1, 3.5; P = .04). There were more mild AE at 4 weeks for the BoNT-A group (P = .002), however, there were no significant between-group differences in the reporting of moderate and serious AE.

Conclusions In a double-blind randomized sham-controlled trial, intramuscular BoNT-A and therapy were effective for improving ease of care and comfort for nonambulant children with CP. There was no increase in moderate and severe AE in the children who had BoNT-A injections compared with the sham group.
Keyword AE, adverse events
BoNT-A, botulinum toxin A
CCHQ, care and comfort hypertonicity questionnaire
COPM, Canadian occupational performance measure
CP, cerebral palsy
CPCHILD, caregiver priorities and child health index of life with disabilities
CPQOL-child, cerebral palsy quality of life questionnaire for children
GEE, generalized estimating equation
GMFCS, gross motor function classification system
PPP, pediatric pain profile
SS, sample size
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ
Additional Notes Available online 12 March 2014

Document type: Journal Article
Sub-type: Article (original research)
Collections: Official 2015 Collection
School of Medicine Publications
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Created: Wed, 19 Mar 2014, 19:37:18 EST by Ms Leanne Sakzewski on behalf of Paediatrics & Child Health - RBWH