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Development, Validation and Evaluation of an Adult and Pediatric Eosinophilic Esophagitis Activity Index: A Prospective Multicenter Study

English title Development, Validation and Evaluation of an Adult and Pediatric Eosinophilic Esophagitis Activity Index: A Prospective Multicenter Study
Applicant Schoepfer Alain
Number 135665
Funding scheme Project funding (Div. I-III)
Research institution Service de Gastroenterologie et Hepatologie CHUV
Institution of higher education University of Lausanne - LA
Main discipline Methods of Epidemiology and Preventive Medicine
Start/End 01.04.2011 - 31.03.2014
Approved amount 331'000.00
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Keywords (7)

eosinophilic esophagitis; activity index; patient reported outcomes; clinical trials; children; adolescents; adults

Lay Summary (German)

Lead
Lay summary

Die eosinophile Speiseröhren-Entzündung (medizinisch "eosinophile Oesophagitis") wurde erstmals 1993 als eigenständige Erkrankung beschrieben. Es handelt sich um eine allergie-artige Entzündung der Speiseröhre (diese stellt die Verbindung her zwischen Mundhöhle und Magen), gekennzeichnet durch eine Ansammlung von eosinophilen Entzündungszellen sowie Schluckbeschwerden oder Brustschmerzen. Im schlimmsten Falle bleibt die Nahrung in der Speiseröhre stecken und erfordert die notfallmässige Entfernung mittels Spiegelung. Unsere Arbeitsgruppe entwickelt ein Instrument um die Entzündungs-Aktivität der Eosinophilen Oesophagitis zu messen (sogenannter Aktivitäts-Index). Dieser Index hat zum Zweck objektive Kriterien für die Erfassung der Entzündungsaktivität zu definieren damit Resultate von verschiedenen Therapie-Studien miteinander verglichen werden können. Wir messen die Aktivität mittels Fragebogen auf den folgenden Ebenen: Beschwerden der Patienten, Aktivität in der Speiseröhrenspiegelung, Aktivität im Gewebe, Entzündung im Blut. Das Projekt hat 3 Phasen: 1. Erstellung der Fragebogen (abgeschlossen), 2. Patienten-Einschluss für Evaluation des Index (aktiv), 3. Patienten-Einschluss für Validierung des Index (nocht nicht begonnen). Das Projekt dauert bis 2014.

Direct link to Lay Summary Last update: 21.02.2013

Responsible applicant and co-applicants

Employees

Publications

Publication
How do gastroenterologists assess overall activity of eosinophilic esophagitis in adult patients?
How do gastroenterologists assess overall activity of eosinophilic esophagitis in adult patients? (2015), How do gastroenterologists assess overall activity of eosinophilic esophagitis in adult patients?, in Am J Gastroenterol., 110, 402-414.
Activity assessment of eosinophilic esophagitis
Schoepfer Alain, Safroneeva Ekaterina (2014), Activity assessment of eosinophilic esophagitis, in Digestive Diseases, 32(1-2), 98-101.
Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis.
Schoepfer AM1 Straumann A2 Panczak R3 Coslovsky M3 Kuehni CE3 Maurer E3 Haas NA3 Romero Y4 H (2014), Development and validation of a symptom-based activity index for adults with eosinophilic esophagitis., in Gastroenterology, 147, 1255-1266.
Eosinophilic esophagitis: overview of clinical management
Schoepfer Alain Hirano Ikuo Katzka David (2014), Eosinophilic esophagitis: overview of clinical management, in Gastroenterol Clin North Am, 43, 329-344.
Eosinophilic oesophagitis and proton pump inhibitor-responsive oesophageal eosinophilia have similar clinical, endoscopic and histological findings.
Moawad Fouad, Schoepfer Alain, Safroneeva Ekaterina, Ally M, Chen Y, Maydanovitch C, Wong R (2014), Eosinophilic oesophagitis and proton pump inhibitor-responsive oesophageal eosinophilia have similar clinical, endoscopic and histological findings., in Alimentary Pharmacology & Therapeutics, 39(6), 603-608.
Swallowed topical corticosteroids reduce the risk for long-lasting bolus impactions in eosinophilic esophagitis.
Kuchen T1 Straumann A Safroneeva E Romero Y Bussmann C Vavricka S Netzer P Reinhard A Portma (2014), Swallowed topical corticosteroids reduce the risk for long-lasting bolus impactions in eosinophilic esophagitis., in Allergy, 69, 1248-1254.
Treatment of eosinophilic esophagitis by dilation.
Schoepfer Alain (2014), Treatment of eosinophilic esophagitis by dilation., in Dig Dis, 32, 130-133.
Treatment of eosinophilic esophagitis by dilation.
Schoepfer Alain (2014), Treatment of eosinophilic esophagitis by dilation., in Digestive Diseases, 32(1-2), 130-133.
Delay in diagnosis of eosinophilic esophagitis increases risk for stricture formation in a time-dependent manner
Schoepfer Alain, Safroneeva Ekaterina, Bussmann Christian, Kuchen Tanja, Portmann Susanne, Simon Hans, Straumann Alex (2013), Delay in diagnosis of eosinophilic esophagitis increases risk for stricture formation in a time-dependent manner, in Gastroenterology, 145(6), 1230-1236.
Eosinophilic esophagitis: the gastroenterologist’s perspective
Schoepfer AM (2012), Eosinophilic esophagitis: the gastroenterologist’s perspective, in Pathologe, 225-227.
Eosinophils in the gastrointestinal tract: friends or foes?
Straumann A Safroneeva E (2012), Eosinophils in the gastrointestinal tract: friends or foes?, in Acta Gastroenterol Belg, 310-315.
Pediatric and adult eosinophilic esophagitis: similarities and differences.
Straumann A Aceves SS Blanchard C Collins MH Furuta GT Hirano I Schoepfer AM Simon D Simon H (2012), Pediatric and adult eosinophilic esophagitis: similarities and differences., in Allergy, 67, 477-490.
Therapeutic concepts in adult and paediatric eosinophilic esophagitis
Straumann A Schoepfer AM (2012), Therapeutic concepts in adult and paediatric eosinophilic esophagitis, in Nat Rev Gastroenterol Hepatol, 9(12), 697-704.
Eosinophilic oesophagitis: latest intelligence
Schoepfer Alain, Straumann Alex (2011), Eosinophilic oesophagitis: latest intelligence, in Clinical & Experimental Allergy, 41(5), 630-639.
Escalating incidence of eosinophilic esophagitis: A 20-year prospective, population-based study in Olten County, Switzerland
Hruz Peter, Straumann Alex, Bussmann Christian, Heer P, Simon H, Zwahlen M, Beglinger Christoph, Schoepfer Alain (2011), Escalating incidence of eosinophilic esophagitis: A 20-year prospective, population-based study in Olten County, Switzerland, in Journal of Allergy and Clinical Immunology, 128(6), 1349-1350.
Update on basic and clinical aspects of eosinophilic oesophagitis
Straumann Alex, Schoepfer Alain, Update on basic and clinical aspects of eosinophilic oesophagitis, in Gut.

Collaboration

Group / person Country
Types of collaboration
Jonathan Spergel, Eosinophilic Disorders, The Children's Hospital of Philadelphia, Philadelphia, PA United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Margaret Collins, Gastro Pathology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Sandeep Gupta, Esophageal Diseases, Riley Hospital for Children, Indianapolis, IN United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Mirna Chehade, Allergic Diseases of GI tract, Mount Sinai Hospital, New York, NY United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Seema Aceves, Eosinophilic Gastrointestinal Disorders Clinic, Rady Children's Hospital, San Diego,CA United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Fouad Moawad, Esophageal Diseases, Walter Reed National Military Medical Center, Bethesda, MD United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Glenn Furuta, Gastrointestinal Eosinophilic Diseases, University of Colorado Denver, Aurora, CO United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Yvonne Romero, Barrett's Esophagus and Esophageal Cancer, Mayo Clinic, Rochester, MN United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Evan Dellon, Esophageal Diseases, University of North Carolina, Chapel Hill, NC United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
John Leung, Eosinophilic Gastrointestinal Diseases, Tufts Medical Center, Boston, MA United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Christian Bussmann, Gastrointestinal Pathology, Viollier AG, Basel Switzerland (Europe)
- in-depth/constructive exchanges on approaches, methods or results
- Publication
Ikuo Hirano, Diseases of the Esophagus, Northwestern University, Chicago, IL United States of America (North America)
- in-depth/constructive exchanges on approaches, methods or results
- Publication

Scientific events

Active participation

Title Type of contribution Title of article or contribution Date Place Persons involved
Digestive Disease Week 2014 Poster Development of a symptom-based activity index for eosinophilic esophagitis 05.05.2014 Chicago, United States of America Zwahlen Marcel; Kühni Claudia; Maurer Schild Elisabeth; Safroneeva Ekaterina; Schoepfer Alain; Panczak Radoslaw; Straumann Alex;
Digestive Disease Week 2014 Poster Development of an endoscopy- and histology-based activity index for eosinophilic esophagitis 05.05.2014 Chicago, United States of America Kühni Claudia; Safroneeva Ekaterina; Schoepfer Alain; Straumann Alex; Maurer Schild Elisabeth; Zwahlen Marcel; Panczak Radoslaw;
United European Gastroenterology Week Poster Duration of untreated inflammation represents the main risk factor for stricture development in eosinophilic esophagitis 15.10.2013 Berlin, Germany Safroneeva Ekaterina; Schoepfer Alain; Straumann Alex;
United European Gastroenterology Week Poster Topical steroid therapy efficiently reduces the risk for long lasting food impactions requiring endoscopic removal in eosinophilic esophagitis 15.10.2013 Berlin, Germany Straumann Alex; Safroneeva Ekaterina; Schoepfer Alain;
United European Gastroenterology Week Talk given at a conference Influence of long-term treatment with topical corticosteroids on natural course of eosinophilic esophagitis and correlation between symptoms and endoscopy, histology, and blood eosinophils 15.10.2013 Berlin, Germany Schoepfer Alain; Safroneeva Ekaterina; Straumann Alex;
Annual congress of the Swiss Society of Gastroenterology Poster Peripheral blood eosinophil counts correlate with esophageal peak eosinophil counts in non-allergic but not in allergic patients with eosinophilic esophagitis 12.09.2013 Basel, Switzerland Straumann Alex; Panczak Radoslaw; Haas Nadine; Safroneeva Ekaterina; Zwahlen Marcel; Kühni Claudia; Schoepfer Alain;
Annual congress of the Swiss Society of Gastroenterology Poster Symptom severity of adult patients with eosinophilic esophagitis measured by the EEsAI PRO module correlates with endoscopic and histologic severity 12.09.2013 Basel, Switzerland Kühni Claudia; Safroneeva Ekaterina; Schoepfer Alain; Panczak Radoslaw; Haas Nadine; Zwahlen Marcel; Straumann Alex;
Annual congress of the Swiss Society of Gastroenterology Poster Development of a standardized method to assess symptoms in adult patients with eosinophilic esophagitis 12.09.2013 Basel, Switzerland Haas Nadine; Schoepfer Alain; Safroneeva Ekaterina; Zwahlen Marcel; Straumann Alex; Kühni Claudia; Panczak Radoslaw;
Digestive Disease Week 2013 Talk given at a conference Influence of long-term treatment with topical corticosteroids on natural course of eosinophilic esophagitis and correlation between symptoms and endoscopy, histology, and blood eosinophils 21.05.2013 Orlando, United States of America Schoepfer Alain; Straumann Alex; Safroneeva Ekaterina;
Digestive Disease Week 2013 Poster Topical steroid theray efficiently reduces the risk for long lasting food impactions requiring endoscopic removal in eosinophilic esophagitis 19.05.2013 Orlando, United States of America Straumann Alex; Safroneeva Ekaterina; Schoepfer Alain;
Digestive Disease Week 2013 Poster Duration of untreated inflammation represents the main risk factor for stricture development in eosinophilic esophagitis 19.05.2013 Orlando, United States of America Safroneeva Ekaterina; Schoepfer Alain; Straumann Alex;
Digestive Disease Week 2013 Poster Eosinophilic esophagitis and proton pump inhibitor responsive esophageal eosinophilia: ar the one and the same? 19.05.2013 Orlando, United States of America Safroneeva Ekaterina; Schoepfer Alain;
Annual congress of the Swiss Society of Gastroenterology Poster Fixed rings and strictures in Eosinophilic Esophagitis develop due to continuing inflammation over time 20.09.2012 Interlaken, Switzerland Schoepfer Alain; Straumann Alex; Safroneeva Ekaterina;
Annual congress of the Swiss Society of Gastroenterology Poster Development of the first disease activity index for eosinophilic esophagitis: update on the EEsAI in 2012 20.09.2012 Interlaken, Switzerland Schoepfer Alain; Kühni Claudia; Zwahlen Marcel; Maurer Schild Elisabeth; Straumann Alex; Safroneeva Ekaterina;
Digestive Disease Week 2012 Individual talk Fixed rings and strictures in Eosinophilic Esophagitis develop due to continuing inflammation over time 22.05.2012 San Diego, United States of America Schoepfer Alain; Straumann Alex; Safroneeva Ekaterina;
Annual congress of the Swiss Society of Gastroenterology Poster The visual dysphagia questionnaire: evaluating the first tool for assessing food consistency-dependent dysphagia in eosinophilic esophagitis 29.09.2011 Lausanne, Switzerland Schoepfer Alain; Safroneeva Ekaterina; Maurer Schild Elisabeth; Kühni Claudia; Straumann Alex; Zwahlen Marcel;
Annual congress of the Swiss Society of Gastroenterology Poster Development of the first disease activity index for eosinophilic esophagitis: update on the EEsAI in 2011 29.09.2011 Lausanne, Switzerland Schoepfer Alain; Safroneeva Ekaterina; Straumann Alex; Zwahlen Marcel; Maurer Schild Elisabeth; Kühni Claudia;
Annual congress of the Swiss Society of Gastroenterology Poster Systematic review of the perforation risk in stenosing eosinophilic esophagitis: dilation is much safer than initially reported 29.09.2011 Lausanne, Switzerland Safroneeva Ekaterina; Straumann Alex; Schoepfer Alain;
Annual congress of the Swiss Society of Gastroenterology Poster How to assess histology in eosinophilic esophagitis: standardization by the EEsAI histopatholgy questionnaire 29.09.2011 Lausanne, Switzerland Maurer Schild Elisabeth; Safroneeva Ekaterina; Straumann Alex; Zwahlen Marcel; Kühni Claudia; Schoepfer Alain;
Digestive Disease Week 2011 Poster Escalating epidemiology of eosinophilic esophagitis: 21 years of prospective population-based documentation in Olten county 07.05.2011 Chicago, United States of America Zwahlen Marcel; Straumann Alex; Schoepfer Alain;


Knowledge transfer events

Active participation

Title Type of contribution Date Place Persons involved
5th PRO Consortium Workshop Workshop 29.04.2014 Silver Springs, United States of America Safroneeva Ekaterina;
Food And Drug Administration, GREAT workshop Talk 19.09.2012 Maryland, Washington, USA, United States of America Schoepfer Alain; Zwahlen Marcel; Straumann Alex; Safroneeva Ekaterina;


Self-organised

Title Date Place
EEsAI Study Update Meeting 2014 05.05.2014 Chicago, United States of America
EEsAI Study Update Meeting 2013 18.05.2013 Orlando, United States of America
EEsAI Study Update Meeting 2012 20.05.2012 San Diego, United States of America
EEsAI Study Update Meeting 2011 08.05.2011 Chicago, United States of America

Communication with the public

Communication Title Media Place Year
Talks/events/exhibitions Soirée d'information pour patients avec oesophagite à éosinophiles, CHUV Western Switzerland 2013

Awards

Title Year
Jahrespreis der schweizerischen Gesellschaft für Allergologie für das paper "development and validation of a symptom-based activity index for adults with eosinopilic esophagitis", Grindelwald, February 6th, 2016 (Auszeichung für Dr. Alain Schoepfer) 2016
Gastroenterologie-Preis der schweizerischen Gesellschaft für Gastroenterologie und Hepatologie für das paper "How do gastroenterologists assess overall activity of eosinophilic esophagitis in adult patients?", Grindelwald, September 2015 (Auszeichnung für Dr. Ekaterina Safroneeva) 2015

Associated projects

Number Title Start Funding scheme
160115 Defining clinically meaningful therapeutic endpoints in Eosinophilic Esophagitis 01.05.2015 Project funding (special)
185008 Development of process-of-care measures and evaluation of their performance in adults with eosinophilic esophagitis (EoE-Q-CARE) 01.03.2020 Project funding (special)

Abstract

Background: Eosinophilic Esophagitis (EoE) is rapidly emerging as a distinctive chronic disorder in pediatric and adult gastroenterology. There is an urgent need for a validated activity index assessing symptoms and the biologic activity of the disease as an objective outcome measure for use in clinical trials, observational studies, and daily clinical practice. Objectives: We will develop and validate a multi-item instrument that can be used to reliably assess the disease activity in adult (ad-EEsAI) and pediatric (ped-EEsAI) EoE patients. Methods: The proposed project involves three distinct phases. While phase 1 is close to being finalized, phases 2 and 3 await completion and are part of this grant application: Phase 1: Selection of candidate items for the index. Item generation (symptoms, biologic markers such as endoscopic findings, histologic and laboratory measures), item reduction and instrument formatting have been achieved through a Delphi process within an international expert group. The goal of this phase was to reach a consensus on relevant items. Phase 2: Derivation of the activity index. In this phase, the item list generated during phase 1 will be evaluated prospectively in a cohort of patients (cohort A) in order to remove unnecessary items and to assess the relative contribution of the remaining items to the overall activity index. Phase 3: Validation of the derived activity index. The methodology of the activity index calculation developed in phase 2 will be validated in a second independent cohort (cohort B). Test-retest reliability and responsiveness of the score will be assessed using longitudinal data from the same patients. Study Population: Two prospective cohorts of adult and pediatric EoE patients will be recruited by collaborating centers in Switzerland, USA and Canada. Cohort A (phase 2) includes 100 adult and 100 pediatric patients, while cohort B (phase 3) includes 200 adult and 200 pediatric patients.Measurements: Physicians will use standardized questionnaires to record symptoms, biologic measures (endoscopic, histologic, and laboratory findings), and treatments of individual patients, and will also be required to provide an overall Physician’s Global Assessment (PGA) of the disease activity.Statistical Analyses: In Phase 2, data will be subjected to a linear regression analysis with the PGA as the primary outcome and variables reflecting the EEsAI items as explanatory variables. In Phase 3, regression coefficients from cohort A will be used to predict the PGA in patients from cohort B, which will then be compared to the recorded PGA provided by the physician. Agreement between predicted and recorded PGA will be analyzed. Test-retest variability and responsiveness to changes in PGA will be assessed using longitudinal data from cohort B. Interobserver variability will be evaluated by comparing 2 independent EEsAI assessments by 2 physicians. Sample size calculations have been performed for Phases 2 and 3. Output and significance: There is a consensus among leading EoE experts that a validated, standardized, and feasible instrument for the assessment of EoE activity is urgently needed in order to conduct prospective randomized clinical trials and natural history studies, as well as for monitoring patients in clinical practice. Therefore, this project represents the unique opportunity to standardize the assessment of EoE activity. The close collaboration with many internationally-recognized EoE centers and the carefully-planned methodology of this study will result in the generation of a widely-accepted disease activity index. Furthermore, recruitment of such large cohorts of patients will allow for a variety of nested projects with particular emphasis on translational research questions relevant to the field of EoE.
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