title: Evaluation of the effects of botulinum toxin A injections when used to improve ease of care and comfort in children with cerebral palsy whom are non-ambulant: a double blind randomized controlled trial.
abstract: BACKGROUND
  Children with cerebral palsy (CP) whom are non-ambulant are at risk of reduced quality of life and poor health status. Severe spasticity leads to discomfort and pain. Carer burden for families is significant. This study aims to determine whether intramuscular injections of botulinum toxin A (BoNT-A) combined with a regime of standard therapy has a positive effect on care and comfort for children with CP whom are non-ambulant (GMFCS IV/V), compared with standard therapy alone (cycle I), and whether repeated injections with the same regime of adjunctive therapy results in greater benefits compared with a single injecting episode (cycle II). The regime of therapy will include serial casting, splinting and/or provision of orthoses, as indicated, combined with four sessions of goal directed occupational therapy or physiotherapy.
  
  
  METHODS
  This study is a double blind randomized controlled trial. Forty participants will be recruited. In cycle I, participants will be randomized to either a treatment group who will receive BoNT-A injections into selected upper and/or lower limb muscles, or a control group who will undergo sham injections. Both groups will receive occupational therapy and /or physiotherapy following injections. Groups will be assessed at baseline then compared at 4 and 16 weeks following injections or sham control. Parents, treating clinicians and assessors will be masked to group allocation. In cycle II, all participants will undergo intramuscular BoNT-A injections to selected upper and/or lower limb muscles, followed by therapy.The primary outcome measure will be change in parent ratings in identified areas of concern for their child's care and comfort, using the Canadian Occupational Performance Measure (COPM). Secondary measures will include the Care and Comfort Hypertonicity Scale (ease of care), the Cerebral Palsy Quality of Life Questionnaire (CP QoL-Child) (quality of life), the Caregiver Priorities and Child Health Index of Life with Disabilities Questionnaire (CPCHILD©) (health status) and the Paediatric Pain Profile (PPP) (pain). Adverse events will be carefully monitored by a clinician masked to group allocation.
  
  
  CONCLUSIONS
  This paper outlines the theoretical basis, study hypotheses and outcome measures for a trial of BoNT-A injections and therapy for children with non-ambulant CP.
  
  
  BACKGROUND
  Australia New Zealand Clinical Trials Registry:N12609000360213.
authors.affiliation: Queensland Cerebral Palsy Health Service, Royal Children Hospital, Brisbane, Australia. m.thorley@uq.edu.au
authors.forename: Megan
authors.initials: M
authors.lastname: Thorley
authors.forename: Samantha
authors.initials: S
authors.lastname: Donaghey
authors.forename: Priya
authors.initials: P
authors.lastname: Edwards
authors.forename: Lisa
authors.initials: L
authors.lastname: Copeland
authors.forename: Megan
authors.initials: M
authors.lastname: Kentish
authors.forename: Kim
authors.initials: K
authors.lastname: McLennan
authors.forename: Jayne
authors.initials: J
authors.lastname: Lindsley
authors.forename: Laura
authors.initials: L
authors.lastname: Gascoigne-Pees
authors.forename: Leanne
authors.initials: L
authors.lastname: Sakzewski
authors.forename: Roslyn N
authors.initials: RN
authors.lastname: Boyd
mesh_terms: D000293:Adolescent; D019274:Botulinum Toxins, Type A / Q000008:administration & dosage*; D002547:Cerebral Palsy / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D004311:Double-Blind Method; D006801:Humans; D007273:Injections, Intramuscular; D009465:Neuromuscular Agents / Q000008:administration & dosage*; D011788:Quality of Life
chemical_list: D009465:Neuromuscular Agents; D019274:Botulinum Toxins, Type A