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