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 alternative to esophageal pneumatic dilatation or surgical myotomy as treatment for achalasia. In the current study, the effects of botulinum toxin were investigated 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 continued to receive botulinum toxin and did not subsequently undergo pneumatic 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 initially 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 surgery, three underwent pneumatic dilatation, eight underwent surgery, three underwent pneumatic dilatation with subsequent surgery, and three awaited surgery. Meta-analysis shows that, in the current study group, the data point expressing time of follow-up evaluation versus percentage of patients needing one injection session without additional procedures (botulinum toxin injection, pneumatic dilatation, or surgery) falls within the curve for those in studies on adult patients receiving botulinum toxin for achalasia.


CONCLUSIONS
Botulinum toxin effectively initiates the resolution of symptoms 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 dilatation or surgery.
Hurwitz
M
M
Department of Pediatrics, University of California Los Angeles School of Medicine, 90095-1752, USA.
Bahar
R J
RJ
Ament
M E
ME
Tolia
V
V
Molleston
J
J
Reinstein
L J
LJ
Walton
J M
JM
Erhart
N
N
Wasserman
D
D
Justinich
C
C
Vargas
J
J
D000293:Adolescent; D000328:Adult; D001905:Botulinum Toxins / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D004931:Esophageal Achalasia / Q000188:drug therapy*; D004947:Esophagus / Q000187:drug effects; D005260:Female; D006801:Humans; D007267:Injections; D008297:Male; D016896:Treatment Outcome
D001905:Botulinum Toxins