Authors' objectives:

This report was prepared in response to a request from the pharmacy of the Montreal Children's Hospital to review the use of botulinum toxin A for the treatment of pediatric patients with chronic anal fissure refractory to other treatments, and for persistent obstructive symptoms (internal anal sphincter achalasia) in children who underwent treatment for Hirschsprung's disease.

Authors' recommendations: Given the sparse evidence of the efficacy and safety of botulinum toxin A in chronic anal fissures and internal sphincter achalasia in pediatric patients, the routine adoption of this technology at this point is not recommended. However, recognizing that definitive studies of rare conditions are unlikely to be available, the important role of clinical acumen, the small budgetary impact of botulinum toxin A, and the possibility that its use may avoid some unnecessary surgery, the Technology Assessment Unit (TAU) recommends that: 1. Botulinum toxin A could be used in the following exceptional circumstances (approximately 4 patients per year), and after consultations with at least two specialists: - In pediatric patients with chronic anal fissures refractory to conservative treatment and who are not eligible for surgery. - In pediatric patients with internal anal sphincter achalasia refractory to conservative treatment, botulinum toxin A could be used as a means of identifying those patients who would benefit from surgical treatment, thus avoiding operating on patients who would likely not benefit from surgery and who might nonetheless be at risk of developing permanent complications. 2. The patients' families should be informed that these are off-label treatment indications for botulinum toxin A that have not been approved by Health Canada. 3. The efficacy and safety outcomes of these patients should be recorded and reviewed in a systematic fashion.

Authors' methods: Review

Project Status: Completed

URL for project: http://upload.mcgill.ca/tau/BOTOX_Dec_27_final.pdf

Year Published: 2005

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Canada

Organisation Name: Technology Assessment Unit of the McGill University Health Centre (MUHC)

Contact Address: Technology Assessment Unit of the MUHC, Centre for Outcomes Research and Evaluation (CORE), Research Institute of the McGill University Health Centre, 5252 boul. de Maisonneuve, Bureau 3F.50, Montreal, Quebec H4A 3S5

Contact Name: nandini.dendukuri@mcgill.ca

Contact Email: nandini.dendukuri@mcgill.ca

Copyright: Technology Assessment Unit of the McGill University Health Centre (MUHC)