- BACKGROUND: Therapeutic options for achalasia include pharmacological therapy, surgical myotomy, pneumatic dilatation and intrasphincteric botulinum toxin injection. AIM: To compare botulinum toxin injection with pneumatic dilatation in a randomized trial. PATIENTS/METHODS: Forty adults with newly diagnosed achalasia were randomized to receive botulinum toxin (n=20) or pneumatic dilatation (n=20). Symptom scores were evaluated at 1, 6 and 12 months. Clinical relapse was defined as a symptom score greater than 50% of baseline. Relapsers received a second botulinum toxin injection or pneumatic dilatation. RESULTS: The cumulative 12-month remission rate was significantly higher after a single pneumatic dilatation (53%) compared to a single botulinum toxin injection (15%)(P < 0.01). The 12-month estimated adjusted hazard for relapse and need for retreatment for the botulinum toxin group was 2.69 times that of the pneumatic dilatation group (95% confidence interval; 1.18-6.14). When a second treatment was administered to the relapsers in each group, the cumulative remission rate 1 year after initial treatment was significantly higher in the pneumatic dilatation group (100%) compared to the botulinum toxin group (60%) (P < 0.01). There were no major complications in either group. CONCLUSIONS: Pneumatic dilatation is more efficacious than botulinum toxin in providing sustained symptomatic relief in patients with achalasia. The efficacy of a single pneumatic dilatation is similar to that of two botulinum toxin injections.