Outcome measures in coeliac disease trials: the Tampere recommendationsCoeliac Disease Centre, Heim Pál Children’s Hospital, Budapest, Hungary; Department of Paediatrics, Faculty of Medicine, University of Debrecen, Debrecen, Hungary.
Celiac Disease Research Center, Faculty of Medicine and Life Sciences, University of Tampere, Tampere, Finland; Department of Paediatrics, Tampere University Hospital, Tampere, Finland.
The Mayo Clinic, Rochester Minnesota, USA.
Institute of Clinical Medicine and K. G. Jebsen Coeliac Disease Research Centre, Faculty of Medicine, University of Oslo, Oslo, Norway; Department of Gastroenterology, Oslo University Hospital, Oslo, Norway.
Science Center, Tampere University Hospital, Tampere, Finland; Tampere Centre for Child Health Research, Faculty of Medicine and Life Sciences, University of Tampere, Tampere, Finland.
Institute for Mother and Child Health Bucharest, University of Medicine and Pharmacy ’Carol Davila’, Bucharest, Romania; Tampere Centre for Child Health Research, University of Tampere, Tampere University Hospital, Tampere,Finland.
Division of Pediatric Gastroenterology, Columbia University Medical Center, New York, USA; Celiac Disease Center, Department of Medicine, Columbia University Medical Center, New York, USA.
Gupo IHP Pediatria, Sevilla, Spain.
Academic Unit of Gastroenterology, Royal Hallamshire Hospital, University of Sheffield, Sheffield, UK.
Celiac Center, University Medical Center, Johannes-Gutenberg University, Mainz, Germany; Division of Gastroenterology, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA.
Coeliac UK, Buckinghamshire, UK.
Tampere Centre for Child Health Research, University of Tampere, Tampere University Hospital, Tampere, Finland.
Continuum Clinical, Northbrook, Illinois, USA.
Faculty of Health and Medicine, School of Medicine and Public Health, University of Newcastle, Newcastle, New South Wales, Australia.
Celiac Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
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2018 (English)In: Gut, ISSN 0017-5749, E-ISSN 1468-3288, Vol. 67, no 8, p. 1410-1424Article in journal (Refereed) Published
Abstract [en]
Objective: A gluten-free diet is the only treatment option of coeliac disease, but recently an increasing number of trials have begun to explore alternative treatment strategies. We aimed to review the literature on coeliac disease therapeutic trials and issue recommendations for outcome measures.
Design: Based on a literature review of 10 062 references, we (17 researchers and 2 patient representatives from 10 countries) reviewed the use and suitability of both clinical and non-clinical outcome measures. We then made expert-based recommendations for use of these outcomes in coeliac disease trials and identified areas where research is needed.
Results: We comment on the use of histology, serology, clinical outcome assessment (including patient-reported outcomes), quality of life and immunological tools including gluten immunogenic peptides for trials in coeliac disease.
Conclusion: Careful evaluation and reporting of outcome measures will increase transparency and comparability of coeliac disease therapeutic trials, and will benefit patients, healthcare and the pharmaceutical industry.
Place, publisher, year, edition, pages
BMJ Publishing Group Ltd, 2018. Vol. 67, no 8, p. 1410-1424
National Category
Gastroenterology and Hepatology
Identifiers
URN: urn:nbn:se:oru:diva-68770DOI: 10.1136/gutjnl-2017-314853ISI: 000442465300008PubMedID: 29440464Scopus ID: 2-s2.0-85047308664OAI: oai:DiVA.org:oru-68770DiVA, id: diva2:1246264
Funder
Swedish Research Council, 522-2A09-195Swedish Society of Medicine
Note
Funding Agencies:
German Research Foundation (DFG)
Ministry of Research and Development (BMBF)
Leibniz Foundation
Biocard
Simtomax
Academy of Finland
Competitive Research Funding of the Tampere University Hospital
Sigrid Juselius Foundation Hungarian Research Fund NKFI 120392
Competitive State Research Financing of the Tampere University Hospital 9U038
2018-09-072018-09-072020-12-01Bibliographically approved