Schoepfer Alain M, Straumann Alex, Panczak Radoslaw, Coslovsky Michael, Kuehni Claudia E, Maurer Elisabeth, Haas Nadine A, Romero Yvonne, Hirano Ikuo, Alexander Jeffrey A, Gonsalves Nirmala, Furuta Glenn T, Dellon Evan S, Leung John, Collins Margaret H, Bussmann Christian, Netzer Peter, Gupta Sandeep K, Aceves Seema S, Chehade Mirna, Moawad Fouad J, Enders Felicity T, Yost Kathleen J, Taft Tiffany H, Kern Emily, Zwahlen Marcel, Safroneeva Ekaterina
Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland.
Division of Gastroenterology and Hepatology, University Hospital Basel, Basel, Switzerland; Swiss Eosinophilic Esophagitis Research Group, Praxis Römerhof, Olten, Switzerland.
Gastroenterology. 2014 Dec;147(6):1255-66.e21. doi: 10.1053/j.gastro.2014.08.028. Epub 2014 Aug 23.
BACKGROUND & AIMS: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE) and to provide end points for clinical trials and observational studies. We aimed to develop and validate a patient-reported outcome (PRO) instrument and score, based on items that could account for variations in patient assessments of disease severity. We also evaluated relationships between patient assessment of disease severity and EoE-associated endoscopic, histologic, and laboratory findings.
We collected information from 186 patients with EoE in Switzerland and the United States (69.4% male; median age, 43 y) via surveys (n = 135), focus groups (n = 27), and semistructured interviews (n = 24). Items were generated for the instruments to assess biologic activity based on physician input. Linear regression was used to quantify the extent to which variations in patient-reported disease characteristics could account for variations in patient assessment of EoE severity. The PRO instrument was used prospectively in 153 adult patients with EoE (72.5% male; median age, 38 y), and validated in an independent group of 120 patients with EoE (60.8% male; median age, 40.5 y).
Seven PRO factors that are used to assess characteristics of dysphagia, behavioral adaptations to living with dysphagia, and pain while swallowing accounted for 67% of the variation in patient assessment of disease severity. Based on statistical consideration and patient input, a 7-day recall period was selected. Highly active EoE, based on endoscopic and histologic findings, was associated with an increase in patient-assessed disease severity. In the validation study, the mean difference between patient assessment of EoE severity (range, 0-10) and PRO score (range, 0-8.52) was 0.15.
We developed and validated an EoE scoring system based on 7 PRO items that assess symptoms over a 7-day recall period. Clinicaltrials.gov number: NCT00939263.
需要标准化工具来评估嗜酸性食管炎（EoE）的活动情况，并为临床试验和观察性研究提供终点指标。我们旨在基于能够解释患者对疾病严重程度评估差异的项目，开发并验证一种患者报告结局（PRO）工具及评分系统。我们还评估了患者对疾病严重程度的评估与EoE相关的内镜、组织学和实验室检查结果之间的关系。
我们通过问卷调查（n = 135）、焦点小组（n = 27）和半结构化访谈（n = 24），收集了来自瑞士和美国的186例EoE患者（69.4%为男性；中位年龄43岁）的信息。根据医生的意见生成用于评估生物活性的工具项目。采用线性回归来量化患者报告的疾病特征差异能够解释患者对EoE严重程度评估差异的程度。该PRO工具前瞻性应用于153例成年EoE患者（72.5%为男性；中位年龄38岁），并在120例独立的EoE患者组（60.8%为男性；中位年龄40.5岁）中进行验证。
用于评估吞咽困难特征、吞咽困难生活行为适应情况以及吞咽时疼痛的7个PRO因素，解释了患者对疾病严重程度评估差异的67%。基于统计学考量和患者反馈，选择了7天的回忆期。基于内镜和组织学检查结果的高度活动性EoE与患者评估的疾病严重程度增加相关。在验证研究中，患者对EoE严重程度的评估（范围0 - 10）与PRO评分（范围0 - 8.52）之间的平均差异为0.15。
我们开发并验证了一种基于7个PRO项目的EoE评分系统，该系统在7天回忆期内评估症状。Clinicaltrials.gov编号：NCT00939263。
