Symptomatic improvement in achalasia after botulinum toxin injection of the lower esophageal sphincter

The aim of this study was to assess the long term clinical outcome of patients with achalasia after treatment with botulinum toxin.

Sixty five patients with achalasia (60 idiopathic, five secondary) were treated with injection of botulinum toxin at the gastroesophageal junction. Dysphagia, chest pain, and regurgitation were scored (0 = no symptoms, 1 = mild, 2 = moderate, 3 = severe, 4 = very severe), with the sum representing the total symptom score, at 0, 7, 30, 120, 240, and 365 days posttreatment. Responders were defined as patients with a 50% decrease in total symptom score at 1 month posttreatment.

The 60 patients with idiopathic achalasia had significant improvement in symptoms of dysphagia, chest pain, and regurgitation at 1 and 4 wk posttreatment. At 1 month posttreatment, 42 of 60 patients (70%) were classified as responders. Of 33 patients with at least 1 yr follow-up, 36% continued to have a good or excellent response, whereas 39% underwent a subsequent treatment with botulinum toxin, pneumatic dilation, or myotomy. When symptoms recurred after an initial response, patients responded to a second injection of botulinum toxin in six of seven cases. In four of five patients with secondary achalasia, there was no response to botulinum toxin.

Botulinum toxin injection at the gastroesophageal junction significantly improved symptoms in 70% of patients with idiopathic achalasia at 1 month. Recurrent symptoms responded to repeat botulinum toxin treatment in initially responsive patients. In contrast, most patients with secondary achalasia did not improve after botulinum toxin injection.