Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)

Ma C, Schoepfer AM, Dellon ES, Bredenoord AJ, Chehade M, Collins MH, Feagan BG, Furuta GT, Gupta SK, Hirano I, Jairath V, Katzka DA, Pai RK, Rothenberg ME, Straumann A, Aceves SS, Alexander JA, Arva NC, Atkins D, Biedermann L, Blanchard C, Cianferoni A, Ciriza de los Rios C, Clayton F, Davis CM, de Bortoli N, Dias JA, Falk GW, Genta RM, Ghaffari G, Gonsalves N, Greuter T, Hopp R, Hsu Blatman KS, Jensen ET, Johnston D, Kagalwalla AF, Larsson HM, Leung J, Louis H, Masterson JC, Menard-Katcher C, Menard-Katcher PA, Moawad FJ, Muir AB, Mukkada VA, Penagini R, Pesek RD, Peterson K, Putnam PE, Ravelli A, Savarino EV, Schlag C, Schreiner P, Simon D, Smyrk TC, Spergel JM, Taft TH, Terreehorst I, Vanuytsel T, Venter C, Vieira MC, Vieth M, Vlieg-Boerstra B, von Arnim U, Walker MM, Wechsler JB, Woodland P, Woosley JT, Yang GY, Zevit N, Safroneeva E (2021)

Publication Type: Journal article

Publication year: 2021

Journal

DOI: 10.1016/j.jaci.2021.07.001

Abstract

Background: End points used to determine treatment efficacy in eosinophilic esophagitis (EoE) have evolved over time. With multiple novel therapies in development for EoE, harmonization of outcomes measures will facilitate evidence synthesis and appraisal when comparing different treatments. Objective: We sought to develop a core outcome set (COS) for controlled and observational studies of pharmacologic and diet interventions in adult and pediatric patients with EoE. Methods: Candidate outcomes were generated from systematic literature reviews and patient engagement interviews and surveys. Consensus was established using an iterative Delphi process, with items voted on using a 9-point Likert scale and with feedback from other participants to allow score refinement. Consensus meetings were held to ratify the outcome domains of importance and the core outcome measures. Stakeholders were recruited internationally and included adult and pediatric gastroenterologists, allergists, dieticians, pathologists, psychologists, researchers, and methodologists. Results: The COS consists of 4 outcome domains for controlled and observational studies: histopathology, endoscopy, patient-reported symptoms, and EoE-specific quality of life. A total of 69 stakeholders (response rate 95.8%) prioritized 42 outcomes in a 2-round Delphi process, and the final ratification meeting generated consensus on 33 outcome measures. These included measurement of the peak eosinophil count, Eosinophilic Esophagitis Histology Scoring System, Eosinophilic Esophagitis Endoscopic Reference Score, and patient-reported measures of dysphagia and quality of life. Conclusions: This interdisciplinary collaboration involving global stakeholders has produced a COS that can be applied to adult and pediatric studies of pharmacologic and diet therapies for EoE and will facilitate meaningful treatment comparisons and improve the quality of data synthesis.

Authors with CRIS profile

Involved external institutions

Northwestern University United States (USA) (US) Nestlé S.A. Switzerland (CH) University of Pennsylvania (UPenn) United States (USA) (US) University of Colorado Anschutz Medical Campus United States (USA) (US) University of Calgary Canada (CA) Indiana University – Purdue University Indianapolis United States (USA) (US) Alimentiv United Kingdom (GB) Universitätsspital Zürich (USZ) Switzerland (CH) Universidad Complutense de Madrid (UCM) Spain (ES) Texas Children's Hospital United States (USA) (US) University of Pisa / Università di Pisa (UniPi) Italy (IT) Hospital de São João / Saint John Hospital Portugal (PT) Inform Diagnostics United States (USA) (US) Penn State College of Medicine United States (USA) (US) Wake Forest University United States (USA) (US) Mayo Clinic in Arizona United States (USA) (US) Huntsman Cancer Institute United States (USA) (US) Lausanne University Hospital / Centre hospitalier universitaire vaudois (CHUV) Switzerland (CH) University of Nebraska Medical Center (UNMC) United States (USA) (US) Ann & Robert H. Lurie Children's Hospital of Chicago United States (USA) (US) Onze Lieve Vrouwe Gasthuis Netherlands (NL) Universitätsklinikum Magdeburg A.ö.R. Germany (DE) University of Newcastle (UoN) Australia (AU) Queen Mary University of London United Kingdom (GB) Schneider Children's Medical Center Israel (IL) Maynooth University Ireland (IE) University of Cincinnati United States (USA) (US) Klinikum rechts der Isar Germany (DE) Mayo Clinic United States (USA) (US) Amsterdam University Medical Centers (Amsterdam UMC) / Amsterdam Universitair Medische Centra Netherlands (NL) University of North Carolina at Chapel Hill United States (USA) (US) Geisel School of Medicine United States (USA) (US) University of Arkansas for Medical Sciences (UAMS) United States (USA) (US) Icahn School of Medicine at Mount Sinai United States (USA) (US) Cincinnati Children's Hospital Medical Center United States (USA) (US) University of California, San Diego (UC San Diego, UCSD) United States (USA) (US) Tufts Medical Center United States (USA) (US) Hôpital Erasme Belgium (BE) Scripps Health United States (USA) (US) Università degli studi di Milano Italy (IT) University of Utah United States (USA) (US) University of Padua / Universita degli Studi di Padova Italy (IT) Inselspital, Universitätsspital Bern Switzerland (CH) University Hospital Leuven (UZ) / Universitaire ziekenhuizen Leuven Belgium (BE) Pontifícia Universidade Católica do Paraná (PUCPR) / Pontifical Catholic University of Paraná Brazil (BR)

How to cite

APA:

Ma, C., Schoepfer, A.M., Dellon, E.S., Bredenoord, A.J., Chehade, M., Collins, M.H.,... Safroneeva, E. (2021). Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS). Journal of Allergy and Clinical Immunology. https://doi.org/10.1016/j.jaci.2021.07.001

MLA:

Ma, Christopher, et al. "Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)." Journal of Allergy and Clinical Immunology (2021).

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