Evaluation of the use of botulinum toxin in children with achalasia

Background: Achalasia is rare in children. Recently, injection of botulinum toxin into the lower esophageal sphincter has been studied as an alternati ve to esophageal pneumatic dilatation or surgical myotomy as treatment for achalasia. In the current study, the effects of botulinum toxin were invest igated in the largest known series of children with achalasia. Methods: Treatment for achalasia was assessed in 23 pediatric patients who received botulinum toxin from June 1995 through November 1998. Those who co ntinued to receive botulinum toxin and did not subsequently undergo pneumat ic dilatation or surgery were considered repeat responders. Results were compared with those of published studies evaluating the use of botulinum toxin in adults with achalasia. Results: Nineteen patients initi ally responded to botulinum toxin. Mean duration of effect was 4.2 months /- 4.0 (SD). At the end of the study period, three were repeat responders, three experienced dysphagia but did not receive pneumatic dilatation or sur gery, three underwent pneumatic dilatation, eight under went surgery, three underwent pneumatic dilatation with subsequent surgery, and three awaited surgery. Meta-analysis shows that, in the current study group, the data poi nt expressing time of follow-up evaluation versus percentage of patients ne eding one injection session without additional procedures (botulinum toxin injection, pneumatic dilatation, or surgery) falls within the curve for tho se in studies on adult patients receiving botulinum toxin for achalasia. Conclusions: Botulinum toxin effectively initiates the resolution of sympto ms associated with achalasia in children. However, one half of patients are expected to need an additional procedure approximately 7 months after one injection session. The authors recommend that botulinum toxin be used only for children with achalasia who are poor candidates for either pneumatic di latation or surgery. (C) 2000 Lippincott Williams & Wilkins, Inc.