TY - JOUR
T1 - EULAR points to consider for conducting clinical trials and observational studies in individuals at risk of rheumatoid arthritis
AU - Mankia, Kulveer
AU - Siddle, Heidi J.
AU - Kerschbaumer, Andreas
AU - Rodriguez, Deshire Alpizar
AU - Catrina, Anca Irinel
AU - Canete, Juan D.
AU - Cope, Andrew P.
AU - Daien, Claire Immediato
AU - Deane, Kevin D.
AU - Gabalawy, Hani El
AU - Finckh, Axel
AU - Holers, V. Michael
AU - Koloumas, Marios
AU - Ometto, Francesca
AU - Raza, Karim
AU - Zabalan, Condruta
AU - van der Helm-van Mil, Annette
AU - van Schaardenburg, Dirkjan
AU - Aletaha, Daniel
AU - Emery, Paul
N1 - Funding Information: Competing interests KM: honoraria from Abbvie, Eli Lilly & Co and UCB; grants from Eli Lilly & Co and Gilead. HJS: none declared. AK: speakers bureau, consultancy: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly & Co, Gilead, Merck Sharp and Dohme, Novartis and Pfizer. DAR: scientific advisor for GSK. AIC, JDC and APC: none declared. CID: honoraria from Roche, Chugai, Pfizer, BMS, MSD, Biogen, Abbvie, Sandoz, Janssen, Novartis, Fresenius Kabi and Sanofi; research grants from Pfizer, MSD and Schwa Medico. KR: speaker fees from Abbvie. PE: expert advice to Pfizer, Abbvie, Amgen, MSD, Roche, Sanofi, BMS, Novartis, Eli Lilly & Co, Gilead, Samsung and Celltrion; grants from Abbvie, Eli Lilly & Co, BMS and Samsung. Funding Information: Funding Funded by EULAR grant CLI 115. Publisher Copyright: © Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. Copyright: Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/10/1
Y1 - 2021/10/1
N2 - Background Despite growing interest, there is no guidance or consensus on how to conduct clinical trials and observational studies in populations at risk of rheumatoid arthritis (RA). Methods An European League Against Rheumatism (EULAR) task force formulated four research questions to be addressed by systematic literature review (SLR). The SLR results informed consensus statements. One overarching principle, 10 points to consider (PTC) and a research agenda were proposed. Task force members rated their level of agreement (1-10) for each PTC. Results Epidemiological and demographic characteristics should be measured in all clinical trials and studies in at-risk individuals. Different at-risk populations, identified according to clinical presentation, were defined: asymptomatic, musculoskeletal symptoms without arthritis and early clinical arthritis. Study endpoints should include the development of subclinical inflammation on imaging, clinical arthritis, RA and subsequent achievement of arthritis remission. Risk factors should be assessed at baseline and re-evaluated where appropriate; they include genetic markers and autoantibody profiling and additionally clinical symptoms and subclinical inflammation on imaging in those with symptoms and/or clinical arthritis. Trials should address the effect of the intervention on risk factors, as well as progression to clinical arthritis or RA. In patients with early clinical arthritis, pharmacological intervention has the potential to prevent RA development. Participants' knowledge of their RA risk may inform their decision to participate; information should be provided using an individually tailored approach. Conclusion These consensus statements provide data-driven guidance for rheumatologists, health professionals and investigators conducting clinical trials and observational studies in individuals at risk of RA.
AB - Background Despite growing interest, there is no guidance or consensus on how to conduct clinical trials and observational studies in populations at risk of rheumatoid arthritis (RA). Methods An European League Against Rheumatism (EULAR) task force formulated four research questions to be addressed by systematic literature review (SLR). The SLR results informed consensus statements. One overarching principle, 10 points to consider (PTC) and a research agenda were proposed. Task force members rated their level of agreement (1-10) for each PTC. Results Epidemiological and demographic characteristics should be measured in all clinical trials and studies in at-risk individuals. Different at-risk populations, identified according to clinical presentation, were defined: asymptomatic, musculoskeletal symptoms without arthritis and early clinical arthritis. Study endpoints should include the development of subclinical inflammation on imaging, clinical arthritis, RA and subsequent achievement of arthritis remission. Risk factors should be assessed at baseline and re-evaluated where appropriate; they include genetic markers and autoantibody profiling and additionally clinical symptoms and subclinical inflammation on imaging in those with symptoms and/or clinical arthritis. Trials should address the effect of the intervention on risk factors, as well as progression to clinical arthritis or RA. In patients with early clinical arthritis, pharmacological intervention has the potential to prevent RA development. Participants' knowledge of their RA risk may inform their decision to participate; information should be provided using an individually tailored approach. Conclusion These consensus statements provide data-driven guidance for rheumatologists, health professionals and investigators conducting clinical trials and observational studies in individuals at risk of RA.
KW - arthritis
KW - autoantibodies
KW - autoimmunity
KW - rheumatoid
UR - http://www.scopus.com/inward/record.url?scp=85112305253&partnerID=8YFLogxK
U2 - https://doi.org/10.1136/annrheumdis-2021-220884
DO - https://doi.org/10.1136/annrheumdis-2021-220884
M3 - Review article
C2 - 34362746
SN - 0003-4967
VL - 80
SP - 1286
EP - 1298
JO - Annals of the rheumatic diseases
JF - Annals of the rheumatic diseases
IS - 10
M1 - 220884
ER -