Use of botulinum toxin as a diagnostic/therapeutic trial to help clarify an indication for definitive therapy in patients with achalasia

OBJECTIVE: Intrasphincteric injection of botulinum toxin is useful in achal asia but is limited by its short term efficacy. The aim of this study was t o evaluate the use of botulinum toxin in selected patients in whom its shor t duration of action may be useful in guiding therapy before considering mo re invasive procedures that might not be indicated. METHODS: Over a 3 yr period, botulinum toxin was injected into the lower es ophageal sphincter in patients with: 1) symptoms consistent with achalasia but insufficient manometric criteria to make the diagnosis; 2) complex clin ical situations in which there were factors in addition to achalasia that m ay be contributing to the patient's symptoms and that required different tr eatment; 3) atypical manifestations of achalasia; 4) advanced achalasia in which it was unclear that sphincter-directed therapy (vs esophagectomy) wou ld be of benefit; and 5) after Heller myotomy. Clinical response was assess ed mostly by symptom improvement, but in some patients follow-up barium swa llow or radioscintigraphy was available. RESULTS: Eleven patients were identified. Ten had complete symptomatic resp onse to the injection. Two patients have undergone subsequent successful pn eumatic dilation, one a successful laparoscopic myotomy, and another curren tly scheduled for surgical myotomy. The only patient without response had a dvanced achalasia requiring esophagectomy. CONCLUSIONS: Intrasphincteric injection of botulinum toxin into the lower e sophageal sphincter is a useful and safe means of guiding therapy in those patients with a variant of achalasia, atypical achalasia, or complex achala sia in which it is unclear that more invasive procedures such as pneumatic dilation or surgical myotomy are the correct therapy. (Am J Gastroenterol 1 999;94:637-642. (C) 1999 by Am. Cell. of Gastroenterology).