Editorials
Selection of study endpoints and patients for clinical trials in primary Sjögren’s syndrome
S. Arends1, L. De Wolff2, L. Deroo3, G.M. Verstappen4, A. Vissink5, F.G. Kroese6, D. Elewaut7, I. Peene8, H. Bootsma9
- Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, The Netherlands. s.arends@umcg.nl
- Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, The Netherlands.
- VIB Centre for Inflammation Research, Ghent University and Department of Rheumatology, Ghent University Hospital, Ghent, Belgium.
- Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, The Netherlands.
- Department of Oral and Maxillofacial Surgery, University of Groningen, University Medical Centre Groningen, The Netherlands.
- Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, The Netherlands.
- VIB Centre for Inflammation Research, Ghent University and Department of Rheumatology, Ghent University Hospital, Ghent, Belgium.
- VIB Centre for Inflammation Research, Ghent University and Department of Rheumatology, Ghent University Hospital, Ghent, Belgium.
- Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Centre Groningen, The Netherlands.
CER15969
2022 Vol.40, N°12
PI 2225, PF 2232
Editorials
Free to view
(click on article PDF icon to read the article)
PMID: 36305355 [PubMed]
Received: 20/06/2022
Accepted : 18/07/2022
In Press: 26/10/2022
Published: 20/12/2022
Abstract
In the last decade, many randomised controlled trials (RCTs) with biological DMARDs (bDMARDs) have been performed in patients with primary Sjögren’s syndrome (pSS). Unfortunately, no bDMARD has yet been approved for systemic treatment of pSS. The heterogeneity of disease manifestations raises two essential questions: 1) which outcome measure is valid, reliable and responsive to demonstrate treatment efficacy and should be used as primary study endpoint? and 2) which pSS patients should be included in clinical trials? Both the selection of the primary study endpoint and the selection of patients are crucial and evolving issues in clinical trial design in pSS. This article summarises the history and comments the selection of primary study endpoints including the novel development of composite endpoints. Furthermore, this article gives an overview of inclusion criteria used for phase II and III trials, and illustrates by data-analysis based on two prospective observational cohorts that each additional selection criterion will (largely) decrease the number of eligible patients in daily clinical practice.
