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 achalasia but is limited by its short term efficacy. The aim of this study was to evaluate the use of botulinum toxin in selected patients in whom its short duration of action may be useful in guiding therapy before considering more invasive procedures that might not be indicated. METHODS: Over a 3 yr period, botulinum toxin was injected into the lower esophageal sphincter in patients with: 1) symptoms consistent with achalasia but insufficient manometric criteria to make the diagnosis; 2) complex clinical situations in which there were factors in addition to achalasia that may be contributing to the patient's symptoms and that required different treatment; 3) atypical manifestations of achalasia; 4) advanced achalasia in which it was unclear that sphincter-directed therapy (vs esophagectomy) would be of benefit; and 5) after Heller myotomy. Clinical response was assessed mostly by symptom improvement, but in some patients follow-up barium swallow or radioscintigraphy was available. RESULTS: Eleven patients were identified. Ten had complete symptomatic response to the injection. Two patients have undergone subsequent successful pneumatic dilation, one a successful laparoscopic myotomy, and another currently scheduled for surgical myotomy. The only patient without response had advanced achalasia requiring esophagectomy. CONCLUSIONS: Intrasphincteric injection of botulinum toxin into the lower esophageal sphincter is a useful and safe means of guiding therapy in those patients with a variant of achalasia, atypical achalasia, or complex achalasia in which it is unclear that more invasive procedures such as pneumatic dilation or surgical myotomy are the correct therapy.