Background and aim: Baclofen, a γ-aminobutyric acid receptor agonist, has been shown to reduce the episodes of gastroesophageal reflux (GER) by reducing the incidence of transient lower esophageal sphincter relaxations. Although baclofen has been shown to reduce reflux symptoms in adults, data in pediatric patients are limited. The aim of the study was to evaluate the efficacy of baclofen in children with refractory GER.
Methods: Medical charts of patients 1 to 18 years of age treated with baclofen for persistent GER symptoms were reviewed retrospectively. Short-term (at first clinic visit) and long-term (12 months) clinical responses were assessed.
Results: A total of 53 patients were included in the final analysis. The mean duration of illness was 1.5 years and the mean age was 6.1 years. All of the patients were taking either once- (53%) or twice-daily (47%) doses of proton pump inhibitors (PPIs) at the time of initiation of baclofen. Thirty-five (66%) patients experienced a significant reduction in clinical symptoms at their first follow-up visit. In the remaining 18 patients, however, baclofen was stopped because of either no response (n = 15) or adverse events (n = 3). A total of 27 patients continued treatment and were assessed for long-term response. Of those, 22 (81%) had a sustained response to baclofen at 12 months, whereas 5 (19%) lost response. We recognized no clinical characteristic differences between those with and without a response to baclofen at either time point.
Conclusions: Baclofen can be used as supplemental therapy to proton pump inhibitors in children with refractory GER; however, prospective trials are needed to further validate our results and assess safety.