Proton Pump Inhibitor Therapy in Pediatric Eosinophilic Esophagitis: Predictive Factors and Long-Term Step-Down Efficacy

To assess the short- and long-term efficacy of a step-down strategy of proton pump inhibitor (PPI) therapy for pediatric eosinophilic esophagitis (EoE) in real world practice and to evaluate possible factors that predict PPI responsiveness.

This study enrolled patients from the nationwide, multicenter, prospective RENSE registry promoted by the Spanish Society for Pediatric Gastroenterology, Hepatology, and Nutrition. Criteria for inclusion in the study included: 1 to 18 years of age, a new diagnosis of EoE, and receiving PPI monotherapy.

This was a cross-sectional study of the prospective RENSE registry. The primary endpoint was histologic remission defined as <15 eosinophils per high powered field (eos/hpf). Additional endpoints included clinical and endoscopic response. Patients were treated based on national consensus guidelines. Transition (step-down) to lower dose PPI therapy occurred following confirmation of histologic remission with initial induction PPI therapy. Multivariate logistic regression models were used to analyze variables associated with histologic remission following PPI induction and maintenance therapy.

Histologic remission following PPI induction therapy was achieved in 51.4% of patients with a median dose of 1.7 mg/kg per day. Clinical and histologic remission was seen in 46.5% of cases. Children with normal endoscopic appearance and lower esophageal eosinophil count at diagnosis were more likely to achieve histologic response after induction therapy, whereas patients with fibrostenotic phenotype at diagnosis were less likely to experience histologic remission. Sustained histologic remission with the first step-down dose (median PPI dose 1.0 mg/kg per day) occurred in 68.5% of patients at a median follow up of 7.2 months. Histologic remission was observed in 85.3% of patients who underwent a second step-down dose (median PPI dose 0.5 mg/kg per day) at a median of 16.4 months. Patients who achieved initial complete histologic remission (<5 eos/hpf) were more likely to have sustained histologic remission. (P < .001)

PPI therapy both induced and sustained histologic remission and symptomatic improvement in pediatric patients with EoE. Patients presenting with the fibrostenotic phenotype were less likely to respond to PPI therapy, whereas those who achieved complete histologic remission with induction PPI therapy were more likely to demonstrate sustained remission.

This study addresses current knowledge gaps regarding PPI therapy for EoE in children and demonstrates that PPI therapy with a step-down dosing strategy is an efficacious option for achieving a durable histologic and clinical remission. Moreover, the identification of factors predicting PPI responsiveness may help to identify good candidates for PPI therapy. This study is limited by its observational nature and underscores the need for randomized controlled trials to further validate the use of PPI therapy in pediatric EoE.