Background. Achalasia is a disorder of swallowing in which the lower e
sophageal sphincter fails to relax, We report the use of botulinum tox
in, a paralytic agent, for the treatment of this condition. Methods. I
n a double-blind trial, 21 patients with achalasia received either 80
units of botulinum toxin or placebo, injected endoscopically into the
lower esophageal sphincter, One week later, the response to treatment
was assessed on the basis of changes in the symptom scores (measured o
n a scale from 0 to 9), pharyngoesophagograms, and results of esophage
al manometric and scintigraphic studies, Patients who received placebo
initially were subsequently treated with botulinum toxin, After six m
onths, esophageal scintigraphy was repeated. Results. One week after t
reatment, the mean decrease in the symptom score was 5.4 points for th
e patients treated with botulinum toxin and 0.5 point for the placebo
group (P=0.001), The mean decrease in the pressure of the lower esopha
geal sphincter was 33 percent in the treatment group, as compared with
a mean increase of 12 percent in the placebo group (P=0.02), and the
mean increase in the width of the opening of the lower esophageal sphi
ncter was 204 percent in the treatment group, as compared with a mean
decrease of 14 percent in the placebo group (P=0.02), Nineteen of the
21 patients treated with botulinum toxin had symptomatic improvement i
nitially; after six months 14 patients were still in remission, This i
mprovement was accompanied by a decrease in esophageal retention that
was sustained at six months (46 percent, as compared with a pretreatme
nt value of 77 percent; P=0.04), There were no serious adverse effects
.Conclusions. Injection of botulinum toxin into the lower esophageal s
phincter is an effective, safe, and simple method of treatment for ach
alasia, with results that are sustained for several months.