Development and Validation of a Symptom-Based Activity Index for Adults With Eosinophilic Esophagitis

被引：216

作者：

Schoepfer, Alain M. ^{[1
]}

Straumann, Alex ^{[2
,3
]}

Panczak, Radoslaw ^{[4
]}

Coslovsky, Michael ^{[4
]}

Kuehni, Claudia E. ^{[4
]}

Maurer, Elisabeth ^{[4
]}

Haas, Nadine A. ^{[4
]}

Romero, Yvonne ^{[5
,6
,7
]}

Hirano, Ikuo ^{[9
]}

Alexander, Jeffrey A. ^{[5
]}

Gonsalves, Nirmala ^{[9
]}

Furuta, Glenn T. ^{[10
]}

Dellon, Evan S. ^{[11
]}

Leung, John ^{[12
,13
]}

Collins, Margaret H. ^{[14
]}

Bussmann, Christian ^{[15
]}

Netzer, Peter ^{[16
]}

Gupta, Sandeep K. ^{[17
]}

Aceves, Seema S. ^{[18
]}

Chehade, Mirna ^{[19
]}

Moawad, Fouad J. ^{[20
]}

Enders, Felicity T. ^{[8
]}

Yost, Kathleen J. ^{[8
]}

Taft, Tiffany H. ^{[9
]}

Kern, Emily ^{[9
]}

Zwahlen, Marcel ^{[4
]}

Safroneeva, Ekaterina ^{[4
]}

机构：

[1] CHU Vaudois, Div Gastroenterol & Hepatol, CH-1011 Lausanne, Switzerland

[2] Univ Basel Hosp, Div Gastroenterol & Hepatol, CH-4031 Basel, Switzerland

[3] Swiss Eosinophil Esophagitis Res Grp, Olten, Switzerland

[4] Univ Bern, Inst Social & Prevent Med, Bern, Switzerland

[5] Mayo Clin, Div Gastroenterol & Hepatol, Rochester, MN USA

[6] Mayo Clin, Dept Otolaryngol, Rochester, MN USA

[7] Mayo Clin, GI Outcomes Unit, Rochester, MN USA

[8] Mayo Clin, Dept Hlth Sci Res, Rochester, MN USA

[9] Northwestern Univ, Feinberg Sch Med, Div Gastroenterol, Chicago, IL 60611 USA

[10] Univ Colorado, Sch Med, Dept Pediat, Aurora, CO USA

[11] Univ N Carolina, Sch Med, Div Gastroenterol & Hepatol, Chapel Hill, NC USA

[12] Tufts Med Ctr, Food Allergy Ctr, Boston, MA USA

[13] Tufts Med Ctr, Div Gastroenterol & Hepatol, Div Allergy & Immunol, Floating Hosp Children, Boston, MA USA

[14] Cincinnati Childrens Hosp Med Ctr, Div Pathol & Lab Med, Cincinnati, OH 45229 USA

[15] Viollier AG, Basel, Switzerland

[16] Lindenhofspital, Div Gastroenterol & Hepatol, Bern, Switzerland

[17] Indiana Univ Sch Med, Riley Hosp Children, Div Pediat Gastroenterol Hepatol & Nutr, Indianapolis, IN 46202 USA

[18] Univ Calif San Diego, Rady Childrens Hosp, Div Allergy & Immunol, San Diego, CA 92103 USA

[19] Mt Sinai Hosp, Mt Sinai Sch Med, Jaffe Food Allergy Inst, Div Gastroenterol, New York, NY 10029 USA

[20] Walter Reed Natl Mil Med Ctr, Gastroenterol Serv, Bethesda, MD USA

来源：

GASTROENTEROLOGY | 2014年 / 147卷 / 06期

基金：

瑞士国家科学基金会;

关键词：

Disease Activity Measurement; Esophagus; Patient-Reported Outcome; Marker; PATIENT-REPORTED OUTCOMES; DYSPHAGIA; BUDESONIDE; ADOLESCENT; GUIDELINES; CHILDREN; CRITERIA;

D O I：

10.1053/j.gastro.2014.08.028

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

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. METHODS: 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). RESULTS: 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. CONCLUSIONS: 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.

引用

页码：1255 / +

页数：33

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