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Gastroenterology. 2014 Dec;147(6):1255-66.e21. doi: 10.1053/j.gastro.2014.08.028. Epub 2014 Aug 23.

Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis.

Author information

1
Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland. Electronic address: alain.schoepfer@chuv.ch.
2
Division of Gastroenterology and Hepatology, University Hospital Basel, Basel, Switzerland; Swiss Eosinophilic Esophagitis Research Group, Praxis Römerhof, Olten, Switzerland. Electronic address: alex.straumann@hin.ch.
3
Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
4
Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota; Department of Otolaryngology, Mayo Clinic, Rochester, Minnesota; GI Outcomes Unit, Mayo Clinic, Rochester, Minnesota.
5
Division of Gastroenterology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
6
Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota.
7
Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colorado.
8
Division of Gastroenterology and Hepatology, University of North Carolina School of Medicine, Chapel Hill, North Carolina.
9
Food Allergy Center at Tufts Medical Center and Floating Hospital for Children, Division of Allergy and Immunology, Division of Gastroenterology and Hepatology, Tufts Medical Center, Boston, Massachusetts.
10
Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
11
Viollier AG, Basel, Switzerland.
12
Division of Gastroenterology and Hepatology, Lindenhofspital, Bern, Switzerland.
13
Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana.
14
Division of Allergy and Immunology, Rady Children's Hospital, University of California, San Diego, San Diego, California.
15
Division of Gastroenterology, Mount Sinai Hospital-Jaffe Food Allergy Institute, Mount Sinai School of Medicine, New York, New York.
16
Gastroenterology Service, Walter Reed National Military Medical Center, Bethesda, Maryland.
17
Department of Health Sciences Research, Mayo Clinic, Rochester, Minnesota.

Abstract

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.

KEYWORDS:

Disease Activity Measurement; Esophagus; Marker; Patient-Reported Outcome

PMID:
25160980
PMCID:
PMC4425255
DOI:
10.1053/j.gastro.2014.08.028
[Indexed for MEDLINE]
Free PMC Article

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