To evaluate the long-term efficacy and safety of proton pump inhibitors (PPIs) in pediatric eosinophilic esophagitis (EoE).

One hundred and nine children between 1 month and 15 years of age with esophageal symptoms and EoE were recruited at 2 pediatric hospitals in Madrid between February 2013 and August 2017. All patients were treated with esomeprazole for 8 weeks, and 72 (66%) achieved histologic remission. Of the 72, 57 children were enrolled in this 1-year study.

This was a prospective study of 57 children (73.7% boys, median age 11 years) with EoE who reached complete or partial histologic remission after an 8-week esomeprazole trial (1 mg/kg per dose, twice daily). Esomeprazole was continued at 1 mg/kg per day (max 40 mg daily) for 1 year. Patients were closely monitored every 3 months for symptom recurrence, clinical outcomes, and adverse events. Follow-up endoscopy was performed at 1 year or before, if symptoms recurred. Complete histologic remission was defined as ≤5 eosinophils per high-power field, and partial remission was defined as >5 and <15 eosinophils per high-power field. Patients were not treated with concomitant dietary restrictions or topical steroids.

After 12 months of PPI maintenance therapy, histologic remission continued in 70.1% (n = 40) of children. Long-term remission was observed more frequently in children with initial complete histologic remission compared with those with partial remission (81% vs 50%, P = .014). There were no other clinical, endoscopic, or histologic data to predict remission on PPI therapy. Eleven of 12 children (91.6%) who remained on esomeprazole at a lower maintenance dose (0.5 mg/kg per day) for an additional 12 months also sustained histologic remission. Five children developed adverse events on esomeprazole (0.7–2 mg/kg per day), including diarrhea, urticaria, and headache. All adverse events self-resolved or improved by substituting for another PPI.

Long-term PPI therapy is safe and effective for the treatment of pediatric EoE. At 1 year follow-up, 70% of children with PPI-responsive EoE remained in histologic and clinical remission on a low-dose maintenance regimen. Patients with complete histologic remission after an 8-week trial of PPI were more likely to maintain histologic remission with maintenance PPI.

This is a landmark prospective study supporting the use of PPI therapy for the long-term treatment of EoE in a subset of pediatric patients. Seventy percent efficacy after 12 months of PPI is comparable or higher than reported remission rates for alternative EoE therapies, such as dietary restriction or swallowed corticosteroids. Future studies will need to compare therapies and determine if PPI dose escalation can lead to improved remission rates.