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

Schoepfer, Alain M; Straumann, Alex; Panczak, Radoslaw; Coslovsky, Michael; Kuehni, Claudia E; Maurer, Elisabeth; Haas, Nadine; 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; ... (2014). Development and Validation of a Symptom-Based Activity Index for Adults with Eosinophilic Esophagitis. Gastroenterology, 147(6), 1255-1266.e21. Elsevier 10.1053/j.gastro.2014.08.028

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BACKGROUND & Aims: Standardized instruments are needed to assess the activity of eosinophilic esophagitis (EoE), to provide endpoints 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 patients' assessments of disease severity. We also evaluated relationships between patients' 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 US (69.4% male; median age, 43 years) via surveys (n = 135), focus groups (n = 27), and semi-structured 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 patients' assessment of EoE severity. The PRO instrument was prospectively used in 153 adult patients with EoE (72.5% male; median age, 38 years), and validated in an independent group of 120 patients with EoE (60.8% male; median age, 40.5 years). 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 patients' 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 and PRO score was 0.13 (on a scale from 0 to 10). CONCLUSIONS We developed and validated an EoE scoring system based on 7 PRO items that assesses symptoms over a 7-day recall period. Clinicaltrials.gov number: NCT00939263.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Pre-clinic Human Medicine > Institute of Social and Preventive Medicine
04 Faculty of Medicine > Pre-clinic Human Medicine > CTU Bern
04 Faculty of Medicine > Department of Gastro-intestinal, Liver and Lung Disorders (DMLL) > Clinic of Visceral Surgery and Medicine > Gastroenterology

UniBE Contributor:

Schöpfer, Alain; Panczak, Radoslaw; Coslovsky, Michael; Kühni, Claudia; Maurer, Elisabeth; Haas, Nadine Agnès Marie-Anne; Zwahlen, Marcel and Safroneeva, Ekaterina

Subjects:

600 Technology > 610 Medicine & health
300 Social sciences, sociology & anthropology > 360 Social problems & social services

ISSN:

0016-5085

Publisher:

Elsevier

Language:

English

Submitter:

Doris Kopp Heim

Date Deposited:

14 Oct 2014 10:38

Last Modified:

24 Aug 2015 02:30

Publisher DOI:

10.1053/j.gastro.2014.08.028

PubMed ID:

25160980

Uncontrolled Keywords:

disease activity measurement esophagus marker patient reported outcome

BORIS DOI:

10.7892/boris.58510

URI:

https://boris.unibe.ch/id/eprint/58510

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