title: Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis.
abstract: OBJECTIVE
  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.
authors.affiliation: Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland. Electronic address: alain.schoepfer@chuv.ch.
authors.forename: Alain M
authors.initials: AM
authors.lastname: Schoepfer
authors.affiliation: 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.
authors.forename: Alex
authors.initials: A
authors.lastname: Straumann
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Radoslaw
authors.initials: R
authors.lastname: Panczak
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Michael
authors.initials: M
authors.lastname: Coslovsky
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Claudia E
authors.initials: CE
authors.lastname: Kuehni
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Maurer
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Nadine A
authors.initials: NA
authors.lastname: Haas
authors.affiliation: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota; Department of Otolaryngology, Mayo Clinic, Rochester, Minnesota; GI Outcomes Unit, Mayo Clinic, Rochester, Minnesota.
authors.forename: Yvonne
authors.initials: Y
authors.lastname: Romero
authors.affiliation: Division of Gastroenterology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
authors.forename: Ikuo
authors.initials: I
authors.lastname: Hirano
authors.affiliation: Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota.
authors.forename: Jeffrey A
authors.initials: JA
authors.lastname: Alexander
authors.affiliation: Division of Gastroenterology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
authors.forename: Nirmala
authors.initials: N
authors.lastname: Gonsalves
authors.affiliation: Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colorado.
authors.forename: Glenn T
authors.initials: GT
authors.lastname: Furuta
authors.affiliation: Division of Gastroenterology and Hepatology, University of North Carolina School of Medicine, Chapel Hill, North Carolina.
authors.forename: Evan S
authors.initials: ES
authors.lastname: Dellon
authors.affiliation: 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.
authors.forename: John
authors.initials: J
authors.lastname: Leung
authors.affiliation: Division of Pathology and Laboratory Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Margaret H
authors.initials: MH
authors.lastname: Collins
authors.affiliation: Viollier AG, Basel, Switzerland.
authors.forename: Christian
authors.initials: C
authors.lastname: Bussmann
authors.affiliation: Division of Gastroenterology and Hepatology, Lindenhofspital, Bern, Switzerland.
authors.forename: Peter
authors.initials: P
authors.lastname: Netzer
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana.
authors.forename: Sandeep K
authors.initials: SK
authors.lastname: Gupta
authors.affiliation: Division of Allergy and Immunology, Rady Children's Hospital, University of California, San Diego, San Diego, California.
authors.forename: Seema S
authors.initials: SS
authors.lastname: Aceves
authors.affiliation: Division of Gastroenterology, Mount Sinai Hospital-Jaffe Food Allergy Institute, Mount Sinai School of Medicine, New York, New York.
authors.forename: Mirna
authors.initials: M
authors.lastname: Chehade
authors.affiliation: Gastroenterology Service, Walter Reed National Military Medical Center, Bethesda, Maryland.
authors.forename: Fouad J
authors.initials: FJ
authors.lastname: Moawad
authors.affiliation: Department of Health Sciences Research, Mayo Clinic, Rochester, Minnesota.
authors.forename: Felicity T
authors.initials: FT
authors.lastname: Enders
authors.affiliation: Department of Health Sciences Research, Mayo Clinic, Rochester, Minnesota.
authors.forename: Kathleen J
authors.initials: KJ
authors.lastname: Yost
authors.affiliation: Division of Gastroenterology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
authors.forename: Tiffany H
authors.initials: TH
authors.lastname: Taft
authors.affiliation: Division of Gastroenterology, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
authors.forename: Emily
authors.initials: E
authors.lastname: Kern
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Marcel
authors.initials: M
authors.lastname: Zwahlen
authors.affiliation: Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland.
authors.forename: Ekaterina
authors.initials: E
authors.lastname: Safroneeva
mesh_terms: D000223:Adaptation, Psychological; D000328:Adult; D003680:Deglutition Disorders / Q000175:diagnosis / Q000473:pathology / Q000503:physiopathology; D016099:Endoscopy, Gastrointestinal; D057765:Eosinophilic Esophagitis / Q000175:diagnosis* / Q000473:pathology* / Q000503:physiopathology; D005247:Feeding Behavior; D005260:Female; D006801:Humans; D016014:Linear Models; D008297:Male; D015203:Reproducibility of Results; D057566:Self Report / Q000592:standards; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires / Q000592:standards*; D013557:Switzerland; D014481:United States
keywords: Disease Activity Measurement; Esophagus; Marker; Patient-Reported Outcome