PATIENT-REPORTED OUTCOME SCORES ARE COMPARABLE IN MULTIREGIONAL VERSUS UNIREGIONAL EOSINOPHILIC ESOPHAGITIS

Gastro hep advances Pub Date : 2025-07-24 DOI:10.1016/j.gastha.2025.100753

Margaret H. Collins MD , Zhaoxing Pan MB, PhD , Guang-Yu Yang MD, PhD , Nicoleta C. Arva MD, PhD , Maria A. Pletneva MD , Lisa J. Martin PhD , Seema Aceves MD, PhD , Mirna Chehade MD, MPH , Evan S. Dellon MD, MPH , Jonathan M. Spergel MD , Paul Menard-Katcher MD , Kathryn A. Peterson MD , Paneez Khoury MD, MHSc , Nirmala Gonsalves MD , Sandeep K. Gupta MD , Carla M. Davis MD , Gary W. Falk MD , Joshua B. Wechsler MD, MSci , Robert Pesek MD , Girish Hiremath MD, MPH , Marc E. Rothenberg MD, PhD

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Abstract

Background and Aims

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

Methods

The Consortium of Eosinophilic Gastrointestinal Researchers database was searched for peak eosinophil counts (PEC), EoE histology scoring system (EoEHSS) scores, and PRO scores (EoE Activity Index, EEsAI; Pediatric EoE Symptom Score, PEESSv2.0; EoE Quality of Life, QoL-A; Pediatric QL EoE modulev3.0, QL) 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.

Results

Adult PEC was 60.2±44.8 vs 39.5±29.9 (mean±SD, 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 QL domain (chest pain/heartburn/stomach aches/nausea/vomiting/food regurgitation) (each P<.043). EoEHSS scores were significantly increased with multi-regional inflammation in adults and children (all P<.01), and scores correlated significantly with symptoms in one uni-regional group (0.29-0.38, P<.01 to .05) but not any multi-regional group.

Conclusions

Multi-regional compared to uni-regional esophageal eosinophil inflammation does not significantly impact most PRO scores, and may not be necessary for all clinical trials.

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患者报告的结果评分在多区域与单区域嗜酸性粒细胞性食管炎中具有可比性

背景和目的嗜酸性粒细胞性食管炎临床试验通常需要多区域食管炎症。为了确定患者报告的预后（PRO）是否因多区域嗜酸性粒细胞炎症（≥15个嗜酸性粒细胞/高倍视野（hpf））而恶化，我们比较了多个（近端/中端/远端）食管区域与一个食管区域炎症时的预后评分。方法在嗜酸性胃肠道研究人员联盟数据库中检索来自一个或多个地区的提交的峰值嗜酸性粒细胞计数（PEC）， EoE组织学评分系统（EoEHSS）评分和PRO评分（EoE活动指数，EEsAI；儿科EoE症状评分，PEESSv2.0； EoE生活质量，QoL-A；儿科QL EoE模块3.0，QL）。对未调整或调整（成人：年龄、性别、1年内扩张；儿童：年龄、性别）数据进行分析，分别采用Wilcoxon秩和、T检验和最小二乘平均值。亚组分析采用交互作用检验。P≤0.05被认为是显著的。结果两个或两个以上部位的成人PEC分别为60.2±44.8 vs 39.5±29.9 (mean±SD, P< 0.004)，儿童PEC分别为66.4±38.2 vs 38.2±31.5 （P=.0007）。当两个或多个区域（67名成人，17名儿童）与一个区域（48名成人，27名儿童）发生炎症时，大多数症状和生活质量评分没有差异：例外是一个成人症状域（调整回避/改变/进食缓慢）和一个儿童症状域（胸痛/胃灼热/胃痛/恶心/呕吐/食物反刍）的适度恶化（每个P<； 0.043）。成人和儿童多区域炎症组EoEHSS评分显著升高（p < 0.01），单区域组EoEHSS评分与症状显著相关（p < 0.01 ~ p < 0.01）。05)，但不是任何多地区集团。结论与单区域食管嗜酸性粒细胞炎症相比，多区域食管嗜酸性粒细胞炎症对大多数PRO评分没有显著影响，可能并非所有临床试验都需要。

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