title: The Effect of Botulinum Toxin A Injections in the Spine Muscles for Cerebral Palsy Scoliosis, Examined in a Prospective, Randomized Triple-blinded Study.
abstract: METHODS
  A prospective, randomized triple-blinded cross-over design treating with either botulinum toxin A (BXT) or saline (NaCl).
  
  
  OBJECTIVE
  To examine the efficacy of BTX treatment in cerebral palsy scoliosis (CPS).
  
  
  BACKGROUND
  Intramuscular injections with BTX have been used off label in treating CPS. 1 prospective study has been conducted, demonstrating in both radiological and clinical improvement, whereas showing no side effects or complications.
  
  
  METHODS
  Subjects (brace-treated CPS between 2 and 18 yr) were injected using ultrasonic-guidance with either NaCl or BTX in selected spine muscles with 6 mo intervals (block randomization, sealed envelope). Radiographs of the spine and clinical follow-up were captured before and 6 weeks after each injection. Primary outcome parameter was radiological change in Cobb angle, where a 7° change was regarded as an effect (1 SD). Radiological parameters were measured before and 6 weeks after treatment by 3 experienced doctors separately. Moreover, clinical results were evaluated by the pediatric quality of life score and systematic open questioning of the parents about the child's wellbeing. Subjects, researchers, and monitors were blinded during the trial. Appropriate permissions (2008-004584-19) and no funding were obtained.
  
  
  RESULTS
  16 cerebral palsy patients (GFMCS III-V) with CPS were consecutively included, whereas 6 patients were excluded. There were no drop-outs to follow-up, but 1 possible serious adverse event of pneumonia resulting in death was recorded and the study was terminated. No significant radiological or clinical changes were detected when compared with NaCl injections using Wilcoxon matched pair signed-rank test.
  
  
  CONCLUSIONS
  No positive radiological or clinical effects were demonstrated by this treatment, except for the parent's initial subjective but positive appraisal of the effect. However, the study was terminated due to 1 possible severe adverse event and scheduled numbers needed to treat (hence power) were not reached.
  
  
  METHODS
  1.
authors.affiliation: *Departments of Orthopaedics †Pediatrics ‡Ambulatory Surgery, and §Centre for Functional and Diagnostic Imaging and Research, Hvidovre University Hospital Denmark.
authors.forename: Christian
authors.initials: C
authors.lastname: Wong
authors.forename: Søren Anker
authors.initials: SA
authors.lastname: Pedersen
authors.forename: Billy B
authors.initials: BB
authors.lastname: Kristensen
authors.forename: Kasper
authors.initials: K
authors.lastname: Gosvig
authors.forename: Stig
authors.initials: S
authors.lastname: Sonne-Holm
mesh_terms: D000293:Adolescent; D019274:Botulinum Toxins, Type A / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D002547:Cerebral Palsy / Q000150:complications*; D002648:Child; D002675:Child, Preschool; D018592:Cross-Over Studies; D005260:Female; D006801:Humans; D007273:Injections, Intramuscular; D008297:Male; D018482:Muscle, Skeletal / Q000187:drug effects / Q000503:physiopathology; D009465:Neuromuscular Agents / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D011446:Prospective Studies; D011859:Radiography; D012600:Scoliosis / Q000188:drug therapy* / Q000209:etiology*; D012965:Sodium Chloride; D013035:Spasm / Q000188:drug therapy*; D013131:Spine / Q000000981:diagnostic imaging / Q000503:physiopathology
chemical_list: D009465:Neuromuscular Agents; D012965:Sodium Chloride; D019274:Botulinum Toxins, Type A