V Navarro-Compán1, A Boel2, A Boonen3, P Mease4, R Landewé5, U Kiltz6, M Dougados7, X Baraliakos6, W Bautista-Molano8, H Carlier9, P Chiowchanwisawakit10, H Dagfinrud11, N de Peyrecave12, B El-Zorkany13, L Fallon14, K Gaffney15, M Garrido-Cumbrera16, L S Gensler17, N Haroon18, Y H Kwan19, P M Machado20, W P Maksymowych21, D Poddubnyy22, M Protopopov23, S Ramiro24, B Shea25, I H Song26, S van Weely27, D van der Heijde2. 1. Rheumatology Service, Hospital Universitario la Paz-IdiPaz, Madrid, Spain. 2. Department of Rheumatology, Leiden University Medical Center, Leiden, the Netherlands. 3. Department of Internal Medicine, Division of Rheumatology, Maastricht University Medical Center, the Netherlands and Care and Public Health Research Institute (CAPHRI), Maastricht University, the Netherlands. 4. Division of Rheumatology, Swedish Medical Center/Providence St. Joseph Health and University of Washington, Seattle, WA, USA. 5. Department of rheumatology & clinical immunology, Amsterdam University Medical Center loc. amC, Amsterdam & Zuyderland MC |loc. Heerlen, The Netherlands. 6. Rheumazentrum Ruhrgebiet Herne, Ruhr-University Bochum, Germany. 7. Université de Paris Department of Rheumatology - Hôpital Cochin. Assistance Publique - Hôpitaux de Paris INSERM (U1153): Clinical epidemiology and biostatistics, PRES Sorbonne Paris-Cité. Paris, France. 8. Rheumatology Department, University Hospital Fundación Santa Fe de Bogotá and School of Medicine Universidad El Bosque. Bogotá, Colombia. 9. Global Clinical Development Immunology, S.A. Eli Lilly Benelux N.V., Brussels, Belgium. 10. Faculty of Medicine Siriraj Hospital, Mahidol University, Thailand. 11. Dept of Rheumatology, Diakonhjemmet Hospital, Oslo, Norway. 12. UBC Pharma, Brussels, Belgium. 13. Rheumatology department, Cairo University. 14. Inflammation and Immunology - Global Medical Affairs, Pfizer Inc, Kirkland, Quebec, Canada. 15. Rheumatology Department, Norfolk & Norwich University Hospitals NHS Foundation Trust, Norwich, UH. 16. Health & Territory Research (HTR), Universidad de Sevilla, Seville, Spain. Spanish Federation of Spondyloartrhtis Associations (CEADE), Madrid, Spain. 17. Division of Rheumatology, Department of Medicine, University of Calfornia, San Francisco, CA, USA. 18. University of Toronto, Departement of Medicine, University Health Network, Schroder Artritis Institute, Toronto. 19. Program in Health Systems and Services Research, Duke-NUS Medical School, Department of Pharmacy, National University of Singapore, Department of Rheumatology and Immunology, Singapore General Hospital. 20. Centre for Rheumatology & Department of Neuromuscular Diseases, University College London, London, United Kingdom; National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre, University College London Hospitals NHS Foundation Trust, London, UK; Department of Rheumatology, Northwick Park Hospital, London North West University Healthcare NHS Trust, London, United Kingdom. 21. Department of Medicine, University of Alberta, Edmonton, Canada. 22. Department of Gastroenterology, Infectious Diseases and Rheumatology, Charité - Universitätsmedizin Berlin, Berlin, Germany. 23. Department of Gastroenterology, Infectiology and Rheumatology, Campus Benjamin Franklin, Charité - Universitätsmedizin Berlin, Germany. 24. Department of Rheumatology, Leiden University Medical Center, Leiden, the Netherlands; Department of Rheumatology, Zuyderland Medical Center, Heerlen, the Netherlands. 25. Ottawa Hospital Research Institute, School of Epidemiology and Public Health, University of Ottawa, ON, Canada. 26. Immunology Clinical Development, 1 North Waukegan Road Building AP31-2, North Chicago, IL 60064, USA. 27. Department of Orthopaedics, Rehabilitation and Physical Therapy, Leiden University Medical Center, Leiden, the Netherlands.
Abstract
BACKGROUND: The current core outcome set for ankylosing spondylitis (AS) has had only minor adaptations since its development 20 years ago. Considering the significant advances in this field during the preceding decades, an update of this core set is necessary. OBJECTIVE: To update the ASAS-OMERACT core outcome set for AS into the ASAS-OMERACT core outcome set for axial spondyloarthritis (axSpA). METHODS: Following OMERACT and COMET guidelines, an international working group representing key stakeholders (patients, rheumatologists, health professionals, pharmaceutical industry and drug regulatory agency representatives) defined the core domain set for axSpA. The development process consisted of: i) Identifying candidate domains using a systematic literature review and qualitative studies; ii) Selection of the most relevant domains for different stakeholders through a 3-round Delphi survey involving axSpA patients and axSpA experts; iii) Consensus and voting by ASAS; iv) Endorsement by OMERACT. Two scenarios are considered based on the type of therapy investigated in the trial: symptom modifying therapies and disease modifying therapies. RESULTS: The updated core outcome set for axSpA includes 7 mandatory domains for all trials (disease activity, pain, morning stiffness, fatigue, physical function, overall functioning and health, and adverse events including death). There are 3 additional domains (extra-musculoskeletal manifestations, peripheral manifestations and structural damage) that are mandatory for disease modifying therapies and important but optional for symptom modifying therapies. Finally, 3 other domains (spinal mobility, sleep, and work and employment) are defined as important but optional domains for all trials. CONCLUSION: The ASAS-OMERACT core domain set for AS has been updated into the ASAS-OMERACT core domain set for axSpA. The next step is the selection of instruments for each domain.
BACKGROUND: The current core outcome set for ankylosing spondylitis (AS) has had only minor adaptations since its development 20 years ago. Considering the significant advances in this field during the preceding decades, an update of this core set is necessary. OBJECTIVE: To update the ASAS-OMERACT core outcome set for AS into the ASAS-OMERACT core outcome set for axial spondyloarthritis (axSpA). METHODS: Following OMERACT and COMET guidelines, an international working group representing key stakeholders (patients, rheumatologists, health professionals, pharmaceutical industry and drug regulatory agency representatives) defined the core domain set for axSpA. The development process consisted of: i) Identifying candidate domains using a systematic literature review and qualitative studies; ii) Selection of the most relevant domains for different stakeholders through a 3-round Delphi survey involving axSpA patients and axSpA experts; iii) Consensus and voting by ASAS; iv) Endorsement by OMERACT. Two scenarios are considered based on the type of therapy investigated in the trial: symptom modifying therapies and disease modifying therapies. RESULTS: The updated core outcome set for axSpA includes 7 mandatory domains for all trials (disease activity, pain, morning stiffness, fatigue, physical function, overall functioning and health, and adverse events including death). There are 3 additional domains (extra-musculoskeletal manifestations, peripheral manifestations and structural damage) that are mandatory for disease modifying therapies and important but optional for symptom modifying therapies. Finally, 3 other domains (spinal mobility, sleep, and work and employment) are defined as important but optional domains for all trials. CONCLUSION: The ASAS-OMERACT core domain set for AS has been updated into the ASAS-OMERACT core domain set for axSpA. The next step is the selection of instruments for each domain.
Authors: Victoria Navarro-Compán; James Cheng-Chung Wei; Filip Van den Bosch; Marina Magrey; Lisy Wang; Dona Fleishaker; Joseph C Cappelleri; Cunshan Wang; Joseph Wu; Oluwaseyi Dina; Lara Fallon; Vibeke Strand Journal: RMD Open Date: 2022-06