Identification of outcome domains in primary Sjögren's disease: A scoping review by the OMERACT Sjögren disease working group.

Sjögren's disease (SjD) is a heterogenous disease with a wide range of manifestations, ranging from symptoms of dryness, fatigue, and pain, to systemic involvement. Considerable advances have been made to evaluate systemic activity or patient-reported outcomes, but most of the instruments were not able to assess all domains of this multifaceted disease. The aim of this scoping review was to generate domains that have been assessed in randomized controlled trials, as the first phase of the Outcome Measures in Rheumatology (OMERACT) process of core domain set development. We systematically searched Medline (Pubmed) and EMBASE between 2002 and March 2023 to identify all randomized controlled trials assessing relevant domains, using both a manual approach and an artificial intelligence software (BIBOT) that applies natural language processing to automatically identify relevant abstracts. Domains were mapped to core areas, as suggested by the OMERACT 2.1 Filter. Among the 5,420 references, we included 60 randomized controlled trials, focusing either on overall disease manifestations (53%) or on a single organ/symptom: dry eyes (17%), xerostomia (15%), fatigue (12%), or pulmonary function (3%). The most frequently assessed domains were perceived dryness (52% for overall dryness), fatigue (57%), pain (52%), systemic disease activity (45%), lacrimal gland function (47%) and salivary function (55%), B-cell activation (60%), and health-related quality of life (40%). Our scoping review highlighted the heterogeneity of SjD, in the study designs and domains. This will inform the OMERACT SjD working group to select the most appropriate core domains to be used in SjD clinical trials and to guide the future agenda for outcome measure research in SjD.

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Copyright © 2024. Published by Elsevier Inc.

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Declaration of competing interest Yann Nguyen, Maxime Beydon, Nathan Foulquier, Rachael Gordon, Coralie Bouillot, and Katherine M. Hammit declared no competing interest. Simon Bowman reports receiving funds for consulting from Bristol-Myers Squibb, Iqvia, Janssen, Kiniksa, Novartis, Otsuka-Visterra. His-salary is part funded by the NIHR Birmingham Biomedical Research centre, Birmingham, UK. Xavier Mariette received consulting fees from Astra-Zeneca, Bristol Myer Squib, Galapagos, GSK, Novartis and Pfizer; travel fees from Novartis. Sara McCoy received consulting fees from BMS, Novartis, Otuska/Visterra, Horizon, Target RWE, Horizon, and Kiniksa. Her time is supported by the Clinical and Translational Science Award (CTSA) program, through the NIH National Center for Advancing Translational Sciences (NCATS), grant 1KL2TR002374 and NIH/NIDCR R03DE031340. Divi Cornec declares no personal financial competing interests and received research funding from Novartis and GSK. Raphaele Seror reports receiving funds for consulting to Bristol-Myers Squibb, Novartis, GSK, Janssen, Amgen, and Boehringher; honoria from Bristol-Myers Squibb, Boehringher, GSK, and travel fees from Amgen and GSK.