Pneumatic balloon dilation in pediatric achalasia: efficacy and factors predicting outcome at a single tertiary pediatric gastroenterology center

Background: The use of pneumatic dilation (PD) is well established in adults with achalasia; however, it is less commonly used in children. Objective: To evaluate the efficacy of PD in pediatric achalasia and to define predictive factors for its treatment failure. Design: Single-center, prospective cohort study. Setting: Academic tertiary referral center. Patients: Twenty-four patients with achalasia were enrolled from January 2004 to November 2009 and were followed for a median of 6 years. Intervention: PD was performed with the patients under general anesthesia. Main outcome measurements: Efficacy and safety of PD. Follow-up was performed by using the Eckardt score, barium swallow contrast studies, and esophageal manometry at baseline; 1, 3, and 6 months after dilation; and every year thereafter. A Cox regression model was used to identify independent predictors of failure after the first PD. Results: The PD success rate was 67%. In 8 patients, the first PD failed, but the parents of one patient refused a second PD and requested surgery. Of the 7 patients who underwent repeated treatment, the second PD failed in 3 (43%). Overall, only 3 of the 24 patients underwent surgery (overall success rate after a maximum of 3 PDs was 87%). Multivariate analysis showed that only older age was independently associated with a higher probability of the procedure success (hazard ratio [HR] 0.66; 95% CI, 0.45-0.97). Limitations: Small sample size, single-center study. Conclusions: PD is a safe and effective technique in the management of pediatric achalasia. Young age is an independent negative predictive factor for successful clinical outcome.