Journal
ANNALS OF THE RHEUMATIC DISEASES
Volume 81, Issue 7, Pages 979-989Publisher
BMJ PUBLISHING GROUP
DOI: 10.1136/annrheumdis-2021-222054
Keywords
Sjogren's syndrome; outcome assessment; health care; patient reported outcome measures
Categories
Funding
- Innovative Medicines Initiative 2 Joint Undertaking (JU) [806975]
- European Union
- EFPIA
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An composite responder index, called Sjogren's Tool for Assessing Response (STAR), was developed in this study to evaluate the treatment response in primary Sjogren's syndrome (pSS). Through data analysis and consensus techniques, the researchers identified five core domains and developed 227 STAR options for sensitivity testing. The candidate STAR showed good sensitivity to change and will be further validated in a dedicated randomized controlled trial.
Objective To develop a composite responder index in primary Sjogren's syndrome (pSS): the Sjogren's Tool for Assessing Response (STAR). Methods To develop STAR, the NECESSITY (New clinical endpoints in primary Sjogren's syndrome: an interventional trial based on stratifying patients) consortium used data-driven methods based on nine randomised controlled trials (RCTs) and consensus techniques involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and the literature, the Delphi panel identified a core set of domains with their respective outcome measures. STAR options combining these domains were proposed to the panel for selection and improvement. For each STAR option, sensitivity to change was estimated by the C-index in nine RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, a meta-analysis of the RCTs was performed. Results The Delphi panel identified five core domains (systemic activity, patient symptoms, lachrymal gland function, salivary gland function and biological parameters), and 227 STAR options combining these domains were selected to be tested for sensitivity to change. After two Delphi rounds, a meta-analysis of the 20 remaining options was performed. The candidate STAR was then selected by a final vote based on metrological properties and clinical relevance. Conclusion The candidate STAR is a composite responder index that includes all main disease features in a single tool and is designed for use as a primary endpoint in pSS RCTs. The rigorous and consensual development process ensures its face and content validity. The candidate STAR showed good sensitivity to change and will be prospectively validated by the NECESSITY consortium in a dedicated RCT.
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