Eligibility for clinical trials in primary Sjögren’s syndrome: lessons from the UK Primary Sjögren’s Syndrome Registry

Abstract

The UKPSSR identified a number of options for trial design, including selection on ESSDAI ⩾5, ESSPRI ⩾5 and serological and other parameters.

Primary Sjögren's Syndrome Prevalence: What if Sjögren was Right After All? Comment on the Article by Maciel et al

“…These results were also in agreement with the clinical impression of trialists with regard to the difficulties of enrolling patients in European multicentric study , and anecdotally with the estimate of 0.05% given in 1933 by Sjögren himself, based on his experience with ophthalmology patients . Importantly, a recent epidemiologic study conducted in the US through the analysis of a US claims database on the 2004–2012 period identified a prevalence of 4 per 10,000 (i.e., 34,473 cases of 84,253 [424 patients]) .…”

Severe Health‐Related Quality of Life Impairment in Active Primary Sjögren's Syndrome and Patient‐Reported Outcomes: Data From a Large Therapeutic Trial

“…Therefore, future therapeutic trials should focus on these symptoms in addition to systemic disease activity. The choice of the primary end point should be made according to the goal of the study and the target population, which are major determinants of the characteristics and feasibility of a trial . Predictive factors of response should be defined for the different drugs studied, and could include general characteristics of the patients but also objective tools such as salivary gland biopsy or major salivary gland ultrasonography .…”

B-cell activity markers are associated with different disease activity domains in primary Sjögren’s syndrome

“…In order to avoid confounding in biomarker studies the ClinESSDAI was developed, in which the biological domain has been removed [ 4 ]. However, the ESSDAI has other limitations, such as its dependence on physician assessment, the difficulty of accurately assessing change in some domains, and the heavy skewing of the population towards low scores [ 5 ]. Therefore, there is interest in developing biomarkers of disease activity such as histopathology [ 6 , 7 ], US [ 8 , 9 ] and serum measures of B-cell activity.…”

“…Understanding the association of biomarkers with specific disease activity domains is important, firstly, to provide insight into the pathogenesis of the differing extra glandular manifestations, and, secondly, to avoid confounding through the expected association of B-cell activity biomarkers with the ESSDAI biological domain or through other domain-specific effects. Therefore, this study aims to investigate the relationship between the B-cell activity biomarkers BAFF, β2M and FLCs with disease activity in a large clinically well-characterized pSS cohort [ 5 ] using the ESSDAI and ClinESSDAI and their individual domains.…”