Abstract

INTRODUCTION: Proton pump inhibitors (PPIs) are now considered a treatment option for eosinophilic esophagitis (EoE). High-dose PPI (HDPPI) response rates are 23-63% in pediatric studies. Our primary aim was to determine PPI response rate in children with EoE. Secondary aim was to determine clinical, endoscopic, or histologic characteristics that impact this PPI response. METHODS: A prospective cohort study of children was conducted. Demographics, clinical history, child and parent-proxy Pediatric EoE Symptom Scores version 2 (PEESSv2.0), EoE endoscopic reference scores (EREFS), and histology scores were collected at endoscopies before and after at least 8 weeks of HDPPI. EoE was defined as ≥ 15 eosinophils/high powered field (eos/hpf) on biopsy from any level of the esophagus on initial endoscopy with symptoms of esophageal dysfunction. Children with < 15 eos/hpf on repeat endoscopy were classified as PPI-responsive (PPI-R) EoE, and ≥ 15 eos/hpf were classified as PPI-nonresponsive (PPI-NR) EoE. RESULTS: 72 children were enrolled from 2015 to 2018 (58% male; mean 9.4 years). 40 children (56%) met criteria for EoE, and 25 had endoscopies before and after HDPPI. 11 refused HDPPI, and 4 were lost to follow-up. 32% (8/25) of children had PPI-R EoE. Baseline demographics, clinical history, child and parent-proxy PEESSv2.0 scores, parent-proxy dysphagia subdomain scores, EREFS, and histology scores were similar in PPI-R and PPI-NR EoE patients. Baseline child dysphagia scores were worse in PPI-NR EoE patients than in PPI-R EoE patients (17.8 v 11.0, P = 0.012). Parent-proxy PEESSv2.0 scores improved in PPI-R and PPI-NR EoE groups after HDPPI (P = 0.046, P = 0.005). Child PEESSv2.0 scores, and parent-proxy and child dysphagia scores improved significantly in the PPI-NR group after HDPPI (33.2 to 19.1, P = 0.006; 18.9 to 8.9, P = 0.004; 17.8 to 9.2, P < 0.001). EREFS improved in PPI-R and PPI-NR EoE groups (P < 0.001, P = 0.030), but had more improvement in the PPI-R group (P = 0.017). Children in the PPI-R group had improvement in degranulation and basal cell hyperplasia compared to the PPI-NR group (P = 0.016, P = 0.008). CONCLUSION: 32% of children with EoE responded to HDPPI therapy. No characteristics predicted PPI response. As anticipated, endoscopic and histologic features of EoE improved more in the PPI-R group. We did not expect to find that child and parent-proxy symptom scores would improve significantly in the PPI-NR group. The value of these symptom scores and their role in EoE management should be further studied.

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