@article{01093cbb4f2245fea62436dc2e50fc9d,
title = "Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis",
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.",
keywords = "Disease Activity Measurement, Esophagus, Marker, Patient-Reported Outcome",
author = "Schoepfer, {Alain M.} and Alex Straumann and Radoslaw Panczak and Michael Coslovsky and Kuehni, {Claudia E.} and Elisabeth Maurer and Haas, {Nadine A.} and Yvonne Romero and Ikuo Hirano and Alexander, {Jeffrey A.} and Nirmala Gonsalves and Furuta, {Glenn T.} and Dellon, {Evan S.} and John Leung and Collins, {Margaret H.} and Christian Bussmann and Peter Netzer and Gupta, {Sandeep K.} and Aceves, {Seema S.} and Mirna Chehade and Moawad, {Fouad J.} and Enders, {Felicity T.} and Yost, {Kathleen J.} and Taft, {Tiffany H.} and Emily Kern and Marcel Zwahlen and Ekaterina Safroneeva and Achem, {Sami R.} and Arora, {Amindra S.} and Oral Alpan and David Armstrong and Attwood, {Stephen E.} and Butterfield, {Joseph H.} and Crowell, {Michael D.} and {De Petris}, Giovanni and DeVault, {Kenneth R.} and Eric Drouin and Benjamin Enav and Fleischer, {David E.} and Amy Foxx-Orenstein and Francis, {Dawn L.} and Guyatt, {Gordon H.} and Harris, {Lucinda A.} and Kagalwalla, {Amir F.} and Katzka, {David A.} and Hirohito Kita and Murli Krishna and Lee, {James J.} and Lewis, {John C.} and Kaiser Lim",
note = "Funding Information: Conflicts of interest Alain Schoepfer has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, Nestl{\'e} SA, and Novartis; he received consulting fees from Aptalis Pharma, Inc, and Dr Falk Pharma, GmbH. Alex Straumann has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, Nestl{\'e} SA, Novartis and Regeneron Pharmaceuticals, Inc; he received consulting fees from Actelion Pharmaceuticals Ltd, B{\"u}hlmann Laboratories AG, Dr Falk Pharma GmbH, Genentech, Inc, Glaxo Smith Kline, plc, Meritage Pharma, Inc, Merck & Co, Inc, Nestle SA, Novartis AG, Oxagen, Ltd, Pfizer, Inc, Receptos, Inc, Regeneron Pharmaceuticals, Inc; he also received speaker fees from Takeda Pharmaceutical Company, Ltd; Radoslaw Panczak has received consulting fees from Aptalis Pharma, Inc; Claudia Kuehni has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, and Nestl{\'e} SA; Yvonne Romero has collaborated on projects supported by Aptalis Pharma, Inc, and Meritage Pharma, Inc, and has received royalties for commercial use of the MDQ-30; Ikuo Hirano has received research grants from Meritage Pharma, Inc, and consulting fees from Aptalis Pharma, Inc, Meritage Pharma, Inc, and Receptos, Inc; Jeffrey Alexander has received consulting fees from Meritage Pharma, Inc, and Aptalis Pharma, Inc, and research grant from Merck & Co, Inc; he has received royalties for commercial use of the MDQ-30, and also has a financial interest in Meritage Pharma, Inc; Glenn Furuta has received consulting fees from Pfizer, Inc, Meritage Pharma, Inc, Knopp, and Biosciences, LLC, and has received royalties from UpToDate, Inc, and is also a founder of EnteroTrack, LLC; Evan Dellon has received research grants from AstraZeneca, AG, and Meritage Pharma, Inc, he has received consulting fees from Aptalis Pharma, Inc, Novartis, Receptos, Inc, and Regeneron Pharmaceuticals, Inc; John Leung has received research grants from Meritage Pharma, Inc; Margaret Collins has received consulting fees from Aptalis Pharma, Inc, Biogen Idec, Meritage Pharma, Inc, Novartis, and Receptos, Inc; Sandeep Gupta has received consulting fees from QOL, Receptos, Inc, and Meritage Pharma, Inc, and speaker fees from Abbott Laboratories and Nestl{\'e} SA. Seema Aceves is a co-inventor of oral viscous budesonide (patent held by University of California, San Diego), and also has received royalties for oral viscous budesonide from Meritage Pharma, Inc, owns stocks in Meritage Pharma, Inc, and has received consulting fees from Receptos, Inc, and Regeneron Pharmaceuticals, Inc; Felicity Enders receives royalties for commercial use of the MDQ-30; Marcel Zwahlen has received research grants from AstraZeneca AG, Aptalis Pharma, Inc, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc, and Nestl{\'e} SA; and Ekaterina Safroneeva has received consulting fees from Aptalis Pharma, Inc, and Novartis. The remaining authors disclose no conflicts. Funding Information: Funding Supported by a grant from the Swiss National Science Foundation ( 32003B_135665/1 ), AstraZeneca AG, Aptalis, Dr Falk Pharma GmbH, Glaxo Smith Kline, plc (MEE114518), Nestl{\'e} SA, Receptos, Inc, and The International Gastrointestinal Eosinophil ResearcherS (A.M.S., A.S., C.E.K., and M.Z.). Also supported by a grant from the National Institute of Health ( 1K24DK100303 to G.T.F.). Publisher Copyright: {\textcopyright} 2014 AGA Institute.",
year = "2014",
month = dec,
day = "1",
doi = "10.1053/j.gastro.2014.08.028",
language = "English (US)",
volume = "147",
pages = "1255--1266.e21",
journal = "Gastroenterology",
issn = "0016-5085",
publisher = "W.B. Saunders Ltd",
number = "6",
}