Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS) - 03/02/22
Doi : 10.1016/j.jaci.2021.07.001
The COREOS Collaborators:
Christopher Ma, MD, MPH a, b, Alain M. Schoepfer, MD c, Evan S. Dellon, MD, MPH d, Albert J. Bredenoord, MD, PhD e, Mirna Chehade, MD, MPH f, Margaret H. Collins, MD g, Brian G. Feagan, MD b, h, i, Glenn T. Furuta, MD j, Sandeep K. Gupta, MD k, Ikuo Hirano, MD l, Vipul Jairath, MD, PhD b, h, i, David A. Katzka, MD m, Rish K. Pai, MD, PhD n, Marc E. Rothenberg, MD, PhD o, Alex Straumann, MD p, Seema S. Aceves, MD, PhD q, Jeffrey A. Alexander, MD m, Nicoleta C. Arva, MD r, Dan Atkins, MD s, Luc Biedermann, MD p, Carine Blanchard, PhD t, Antonella Cianferoni, MD, PhD u, Constanza Ciriza de los Rios, MD v, Frederic Clayton, MD w, Carla M. Davis, MD x, Nicola de Bortoli, MD y, Jorge A. Dias, MD z, Gary W. Falk, MD, MS aa, Robert M. Genta, MD bb, cc, Gisoo Ghaffari, MD dd, Nirmala Gonsalves, MD l, Thomas Greuter, MD c, p, Russell Hopp, DO ee, Karen S. Hsu Blatman, MD ff, Elizabeth T. Jensen, MPH, PhD gg, Doug Johnston, MD hh, Amir F. Kagalwalla, MD ii, jj, Helen M. Larsson, MD, PhD kk, John Leung, MD, PhD ll, Hubert Louis, MD mm, Joanne C. Masterson, PhD nn, Calies Menard-Katcher, MD j, Paul A. Menard-Katcher, MD oo, Fouad J. Moawad, MD pp, Amanda B. Muir, MD qq, Vincent A. Mukkada, MD rr, Roberto Penagini, MD ss, tt, Robert D. Pesek, MD uu, Kathryn Peterson, MD vv, Philip E. Putnam, MD rr, Alberto Ravelli, MD ww, Edoardo V. Savarino, MD, PhD xx, Christoph Schlag, MD, PhD yy, Philipp Schreiner, MD p, Dagmar Simon, MD zz, Thomas C. Smyrk, MD aaa, Jonathan M. Spergel, MD, PhD u, Tiffany H. Taft, PsyD l, Ingrid Terreehorst, MD, PhD bbb, Tim Vanuytsel, MD ccc, ddd, Carina Venter, PhD, RD s, Mario C. Vieira, MD, PhD eee, Michael Vieth, MD fff, Berber Vlieg-Boerstra, MD ggg, Ulrike von Arnim, MD hhh, Marjorie M. Walker, BMBS, FRCPath iii, Joshua B. Wechsler, MD, MS ii, Philip Woodland, MD jjj, John T. Woosley, MD kkk, Guang-Yu Yang, MD, PhD lll, Noam Zevit, MD mmm, nnn, Ekaterina Safroneeva, PhD ooo, ⁎
a Division of Gastroenterology and Hepatology, Departments of Medicine and Community Health Sciences, University of Calgary, Calgary, Alberta, Canada
b Alimentiv Inc, London, Ontario, Canada
c Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois (CHUV) and University of Lausanne, Lausanne, Switzerland
d Center for Esophageal Diseases and Swallowing, Division of Gastroenterology and Hepatology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC
e Department of Gastroenterology and Hepatology, Amsterdam University Medical Center, Amsterdam, The Netherlands
f Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY
g Division of Pathology and Laboratory Medicine, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
h Department of Medicine, Western University, London, Ontario, Canada
i Department of Epidemiology and Biostatistics, Western University, London, Ontario, Canada
j Digestive Health Institute, Children’s Hospital Colorado, Gastrointestinal Eosinophilic Diseases Program, Section of Pediatric Gastroenterology, Hepatology, and Nutrition, University of Colorado School of Medicine, Aurora, Colo
k Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Riley Hospital for Children/Indiana University School of Medicine, Indianapolis, Ind
l Division of Gastroenterology & Hepatology, Northwestern University, Feinberg School of Medicine, Chicago, Ill
m Division of Gastroenterology, Mayo Clinic, Rochester, Minn
n Department of Pathology and Laboratory Medicine, Mayo Clinic Arizona, Scottsdale, Ariz
o Division of Allergy and Immunology, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio
p Department of Gastroenterology and Hepatology, University Hospital Zurich, Zurich, Switzerland
q Division of Allergy Immunology, University of California, San Diego, Rady Children’s Hospital, San Diego, Calif
r Department of Pathology and Laboratory Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Feinberg School of Medicine, Chicago, Ill
s Gastrointestinal Eosinophilic Diseases Program, Children’s Hospital of Colorado, Section of Allergy and Immunology, University of Colorado School of Medicine, Aurora, Colo
t Nestlé Institute of Health Sciences, Nestlé Research, Société des Produits Nestlé, Vevey, Switzerland
u Division of Allergy and Immunology, Department of Pediatrics, Children’s Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania, Philadelphia, Pa
v Department of Gastroenterology, Hospital Clínico San Carlos, Universidad Complutense, Instituto de Investigación Sanitaria San Carlos (IdISSC), Madrid, Spain
w Department of Pathology, The University of Utah, Huntsman Cancer Hospital, Salt Lake City, Utah
x Immunology, Allergy, and Retrovirology Section of the Department of Pediatrics, Baylor College of Medicine, Texas Children’s Hospital, Houston, Tex
y Department of Translational Research and New Technology in Medicine and Surgery, Division of Gastroenterology, University of Pisa, Cisanello Hospital, Pisa, Italy
z Pediatric Gastroenterology, Centro Hospitalar S. João, Porto, Portugal
aa Division of Gastroenterology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pa
bb Inform Diagnostics, Irving, Tex
cc Department of Pathology, Baylor College of Medicine, Houston, Tex
dd Division of Pulmonary, Allergy, and Critical Care Medicine, Department of Medicine, Pennsylvania State College of Medicine, Hershey, Pa
ee University of Nebraska Medical Center, Children’s Hospital and Medical Center, Omaha, Neb
ff Section of Allergy and Clinical Immunology, Dartmouth-Hitchcock Medical Center, Dartmouth Geisel School of Medicine, Hanover, NH
gg Wake Forest University School of Medicine, Department of Epidemiology and Prevention, Winston-Salem, NC
hh Asthma and Allergy Specialists, Charlotte, NC
ii Division of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, Ill
jj Division of Gastroenterology, Department of Pediatrics, John H. Stroger Jr Hospital of Cook County, Chicago, Ill
kk Department of ENT, Head, and Neck Surgery, NÄL Medical Centre, Trollhättan, Sweden
ll Division of Gastroenterology, Tufts Medical Center, Boston, Mass
mm Department of Gastroenterology, Hepatopancreatology, and Digestive Oncology, Erasme Hospital, Université Libre de Bruxelles, Brussels, Belgium
nn Department of Biology, Maynooth University, Kildare, Ireland
oo Division of Gastroenterology and Hepatology, University of Colorado Anschutz Medical Campus, Aurora, Colo
pp Division of Gastroenterology & Hepatology, Scripps Clinic, La Jolla, Calif
qq Center for Pediatric Eosinophilic Diseases, Division of Gastroenterology and Hepatology & Nutrition, Children’s Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pa
rr Division of Gastroenterology, Hepatology, and Nutrition, Cincinnati Children’s Hospital Medical Center, Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio
ss Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy
tt Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy
uu Division of Allergy and Immunology, University of Arkansas for Medical Sciences and Arkansas Children’s Hospital, Little Rock, Ark
vv Division of Gastroenterology, The University of Utah, Salt Lake City, Utah
ww University Department of Pediatrics, Children’s Hospital–Spedali Civili, Brescia, Italy
xx Department of Surgery, Oncology, and Gastroenterology, DiSCOG, University of Padua, Padua, Italy
yy II. Medizinische Klinik, Klinikum Rechts der Isar, Technische Universität München, Munich, Germany
zz Department of Dermatology, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
aaa Department of Pathology, Mayo Clinic, Rochester, Minn
bbb Department of ENT, Amsterdam University Medical Centre, Amsterdam, The Netherlands
ccc Gastroenterology and Hepatology, University Hospitals Leuven, Leuven, Belgium
ddd Translational Research in Gastrointestinal Disorders, KU Leuven, Leuven, Belgium
eee Department of Pediatrics, Pontifical Catholic University of Paraná and Center for Pediatric Gastroenterology, Hospital Pequeno Príncipe, Curitiba, Brazil
fff Institute for Pathology, Klinikum Bayreuth, Friedrich-Alexander-University Erlangen-Nuremberg, Erlangen, Germany
ggg OLVG Hospital, Department of Pediatrics, Amsterdam, The Netherlands
hhh Department of Gastroenterology, Hepatology, and Infectious Diseases, University Hospital, Magdeburg, Germany
iii Centre of Research Excellence in Digestive Health, University of Newcastle, Newcastle, Australia
jjj Wingate Institute of Neurogastroenterology, Barts, and the London School of Medicine and Dentistry, Queen Mary University of London, London, England, United Kingdom
kkk Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC
lll Division of Pathology, Northwestern University, Feinberg School of Medicine, Chicago, Ill
mmm Institute of Gastroenterology, Nutrition, and Liver Diseases, Schneider Children’s Medical Center of Israel, Petach Tikva, Israel
nnn Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
ooo Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland
∗Corresponding author: Ekaterina Safroneeva, PhD, Institute of Social and Preventive Medicine, University of Bern, Mittelstrasse 43, Bern 3012, Switzerland.Institute of Social and Preventive MedicineUniversity of BernMittelstrasse 43Bern3012Switzerland
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.
Le texte complet de cet article est disponible en PDF.Key words : Eosinophilic esophagitis, outcomes, clinical trials, end points, histology, histopathology, endoscopy, symptoms, patient-reported outcomes, quality of life
Abbreviations used : AAAAI, CEGIR, COMET, COREOS, COS, DSQ, EEsAI, EGID, EoE, EoEHSS, EREFS, EUREOS, FDA, hpf, PEC, PedsQL, PEESS, PRO, QoL, RCT
Plan
| The first 2 authors contributed equally to this article, and both should be considered first author. |
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| Supported by grants from the Swiss National Science Foundation (32003B_160115/1 to A.M.S. and 32473B_185008 to E.S.), Switzerland. |
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| Disclosure of potential conflict of interest: C. Ma reports consulting fees from AVIR Pharma and Alimentiv (formerly Robarts Clinical Trials). A. M. Schoepfer reports consulting fees, speaker fees, and/or research grants from Adare Pharmaceuticals, AstraZeneca (Switzerland), Aptalis Pharma, Celgene, Dr Falk Pharma (Germany), GlaxoSmithKline, Nestlé (Switzerland), Novartis (Switzerland), Receptos, and Regeneron Pharmaceuticals. E. S. Dellon reports research funding from Adare/Ellodi, Allakos, AstraZeneca, GSK, Meritage, Miraca, Nutricia, Celgene/Receptos/BMS, Regeneron, Shire/Takeda; consulting fees from Abbott, Adare/Ellodi, Aimmune, Allakos, Amgen, Arena, AstraZeneca, Avir, Biorasi, Calypso, Celgene/Receptos/BMS, Celldex, Eli Lilly, EsoCap, GSK, Gossamer Bio, Parexel, Regeneron, Alimentiv, Salix, Sanofi, and Shire/Takeda; and educational grants from Allakos, Banner, and Holoclara. A. J. Bredenoord reports research funding from Nutricia, Norgine, SST, and Bayer; and received speaker and/or consulting fees from Laborie, Arena, EsoCap, Medtronic, Dr Falk Pharma, Calypso Biotech, Gossamer, Alimentiv, Reckett Benkiser, Regeneron, and AstraZeneca. M. Chehade reports research funding from Regeneron, Allakos, Shire, AstraZeneca, and Danone; consulting fees from Regeneron, Allakos, Adare, Shire/Takeda, AstraZeneca, Sanofi, and Bristol Myers Squibb; and lecture honoraria from Nutricia, Medscape, and Vindico. M. H. Collins is a consultant for Allakos, Arena, Astra Zeneca, Calypso, Esocap, GSK, Receptos/BMS, Regeneron, Shire/Takeda, and Alimentiv (formerly Robarts Clinical Trials); and reports research grants from Receptos/BMS, Regeneron, and Shire/Takeda. B. G. Feagan reports consulting fees from Allakos, Alimentiv (formerly Robarts Clinical Trials), Sanofi, and Bristol Myers Squibb. G. T. Furuta reports salary support from EnteroTrack. S. K. Gupta reports personal fees from Allakos, Abbott, Adare, Celgene, Gossamer Bio, QOL, Medscape, and Viaskin; research grants from Shire; and royalties from UpToDate. I. Hirano reports research funding from Adare, Allakos, GSK, Meritage, Celgene/Receptos, Regeneron, and Shire/Takeda; and consulting fees from Adare, Allakos, Arena, AstraZeneca, Celgene/Receptos, Eli Lilly, EsoCap, GSK, Gossamer Bio, Regeneron, and Shire/Takeda. V. Jairath reports consulting fees from Alimentiv (formerly Robarts Clinical Trials). D. A. Katzka reports consulting fees from Takeda, Sanofi, and Shire. R. K. Pai reports consulting fees from Eli Lilly, Genentech, Allergan, and Alimentiv (formerly Robarts Clinical Trials). M. E. Rothenberg reports personal fees from Celgene, Astra Zeneca, Arena Pharmaceuticals, Adare Pharmaceuticals, GlaxoSmithKline, Guidepoint, and Suvretta Capital Management; has an equity interest in Pulm One, Spoon Guru, ClostraBio, Serpin Pharm, and Allakos; received royalties from Teva Pharmaceuticals (for reslizumab), Mapi Research Trust (for PEESSv2), and UpToDate; and is an inventor of patents owned by Cincinnati Children’s Hospital. A. Straumann reports personal fees from Allakos, Astra-Zeneca, Calypso, EsoCap, Dr Falk Pharma, Gossamer, Nutricia, Pfizer, Receptos-Celgene, Regeneron-Sanofi, Roche-Genentec, Shire, and Tillotts. S. S. Aceves reports being a consultant for Regeneron, Astra-Zeneca, Astellos, and AImmune; and has a UCSD patent licensed to Shire/Takeda Pharma. J. A. Alexander reports personal fees or grants from Regeneron and Adare Pharmaceuticals and has equity interest in Meritage Pharmacia. L. Biedermann reports personal fees from Vifor, Dr Falk Pharma, Esocap, and Calypso. C. Blanchard is an employee of Société des produits Nestlé. C. Ciriza de los Rios reports consulting fees for Norgine and Allergan. C. M. Davis reports research grants from the National Institutes of Health/National Institute of Allergy and Infectious Disease (Consortium of Food Allergy Research/Consortium of Eosinophilic Gastrointestinal researchers), DBV Technologies, Aimmune Therapeutics, and Regeneron Pharmaceuticals; and owns stock in Moonlight Therapeutics. G. W. Falk reports grants and/or personal fees from Allakos, Shire/Takeda, ADARE/Ellodi, Regeneron, and Bristol Myers Squibb. R. M. Genta reports consulting fees from Allakos, Adare/Ellodi, and RedHill Pharma. N. Gonsalves reports consulting fees from Allakos, Astra-Zeneca, Nutricia, and Sanofi/Regeneron; and royalties from UpToDate. T. Greuter reports consulting contracts with Dr Falk Pharma and Sanofi-Aventis, and a research grant from Novartis. K. S. Hsu Blatman reports research funding from Shire/Takeda. H. M. Larsson reports consulting fees from EsoCap Biotech. F. J. Moawad reports personal fees from Takeda and Salix. A. V. A. Mukkada reports grants and/or personal fees from Shire Pharmaceutical. K. Peterson reports personal fees from Alladapt, Eli Lily, Medscape, Ellodi, Takeda, Allakos, AstraZeneca, and Regeneron-Sanofi; research funding or other research support from Astra Zeneca, Ellodi, Regeneron-Sanofi, Allakos, and Chobani; and owns stock in Nexeos. C. Schlag reports consulting fees, speaker fees, and/or research grants from Adare Pharmaceuticals, AstraZeneca, Calypso, EsoCap, Dr Falk Pharma, and Regeneron Pharmaceuticals. P. Schreiner reports consulting fees from Pfizer, Takeda, and Janssen-Cilag. T. J. M. Spergel reports grants and/or personal fees from DBV Technologies, End Allergy Together, Food Allergy Research Education, Aimmune Therapeutics, UpToDate, Regeneron, and Shire. T. H. Taft reports speaking fees from AbbVie and consulting fees from Healthline. T. Vanuytsel has served as a speaker for Abbott, Dr Falk Pharma, Fresenius Kabi, Kyowa Kirin and Menarini, Takeda, and Will Pharma; has served as a consultant and advisory board member for Baxter, Dr Falk Pharma, Takeda, Tramedico, Truvion, VectivBio, and Zealand Pharma; and has received research funding from Danone and MyHealth. C. Venter has provided and reviewed educational material for Danone, Reckitt Benckiser, Abbott Nutrition, DBV Technologies, and Nestlé Nutrition Institute; and has received research grants from the National Peanut Board and Reckitt Benckiser. M. C. Vieira reports speaker’s fees from Danone Nutricia and Nestlé. M. Vieth reports speaker’s fees from Dr Falk Pharma, Shire, and Menarini. U. von Arnim reports consulting fees from ESOCAP, AbbVie, MSD, Takeda, and Dr Falk Pharma. J. B. Wechsler reports consulting fees from Allakos and Regeneron. N. Zevit reports speaker’s fees for Dr Falk Pharma; and is an advisory board member for Adare Pharmaceuticals. E. Safroneeva reports consulting fees from AVIR Pharma, Aptalis Pharma, Celgene, Novartis, and Regeneron Pharmaceuticals. The following authors declare that they have no relevant conflicts of interest: N. de Bortoli, D. Johnston, A. F. Kagalwalla, A. Ravelli, and B. Vlieg-Boerstra. |
© 2021
The Authors. Publié par Elsevier Masson SAS. Tous droits réservés.
Vol 149 - N° 2
P. 659-670 - février 2022 Retour au numéro
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