Patient-Reported Outcome Scores Are Comparable in Multiregional Versus Uniregional Eosinophilic Esophagitis

Eosinophilic esophagitis (EoE) clinical trials often require multiregional esophageal inflammation. To determine if patient-reported outcomes (PROs) worsen with multiregional eosinophil inflammation (≥15 eosinophils/high power field), we compared outcomes scores when multiple (proximal/mid/distal) esophageal regions, versus one, were inflamed.

The Consortium of Eosinophilic Gastrointestinal Disorders Researchers database was searched for peak eosinophil counts (PECs), eosinophilic esophagitis histology scoring system scores, and PRO scores (eosinophilic esophagitis activity index; Pediatric Eosinophilic Esophagitis Symptom Score; EoE quality of life; Pediatric Quality of Life Eosinophilic Esophagitis modulev3.0) in submissions from one or more regions. Analyses were performed with unadjusted or adjusted (adults: age, sex, dilation within 1 year; children: age, sex) data, using Wilcoxon rank sum and T test, and least squares mean, respectively. An interaction test was used for subgroup analysis. P ≤ .05 was considered significant.

Adult PEC was 60.2 ± 44.8 vs 39.5 ± 29.9 (mean ± standard deviation, P < .004), child PEC was 66.4 ± 38.2 vs 38.2 ± 31.5 (P = .0007) when two or more regions, vs one, were inflamed, respectively. Most symptoms and QoL scores did not differ when two or more regions (67 adults, 17 children) vs one (48 adults, 27 children) were inflamed: exceptions were modest worsening of one adult symptom domain (adjusted avoidance/modification/slow eating), and one child Pediatric QL Eosinophilic Esophagitis modulev3.0 domain (chest pain/heartburn/stomach aches/nausea/vomiting/food regurgitation) (each P < .043). Eosinophilic esophagitis histology scoring system scores were significantly increased with multiregional inflammation in adults and children (all P < .01), and scores correlated significantly with symptoms in one uniregional group (0.29-0.38, P < .01 to 0.05) but not any multiregional group.

Multiregional compared to uniregional esophageal eosinophil inflammation does not significantly impact most PRO scores, and may not be necessary for all clinical trials.