Seror, Raphaele, Baron, Gabriel, Camus, Marine, Cornec, Divi, Perrodeau, Elodie, Bowman, Simon, Bombardieri, Michele, Bootsma, Hendrika, Gottenberg, Jaccques-Eric, Fisher, Benjamin, Hueber, Wolfgang, van Roon, Joel, Devauchelle-Pensec, Valerie, Gergely, Peter, Mariette, Xavier and Porcher, Raphael (2022) Development and preliminary validation of the Sjögren’s Tool for Assessing Response (STAR): a consensual composite score for assessing treatment effect in primary Sjögren’s syndrome. Annals of the rheumatic diseases : ARD, Online (Online). Online-Online. ISSN Online ahead of print

Abstract

Objectives:
To develop a composite responder index in primary Sjögren’s syndrome (pSS): the Sjögren’s Tool for Assessing Response (STAR).
Methods:
To develop STAR, the NECESSITY consortium used data-driven methods, based on 9 randomized controlled trials (RCTs), and consensus techniques, involving 78 experts and 20 patients. Based on reanalysis of rituximab trials and 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 to select and improve them. For each STAR option, sensitivity to change was estimated by the C-index in 9 RCTs. Delphi rounds were run for selecting STAR. For the options remaining before the final vote, meta-analyses of the RCTs were performed.
Results:
The Delphi panel identified 5 domains (systemic activity, patient symptoms, lachrymal function, salivary gland function and biological parameters). A total of 227 STAR options, combining these domains, were selected to be tested for sensitivity to change in the 9 RCTs. After 2 Delphi rounds, meta-analyses of the 20 remaining options were performed. Then, the candidate STAR was selected by a final vote.
Conclusion:
The candidate STAR is a composite responder index, including in a single tool all main disease features, and dedicated for use as a primary endpoint in pSS RCTs. Its rigorous and consensual development process ensures its face and content validity. The candidate STAR showed a good sensitivity to change. The candidate STAR and its derived options will be prospectively validated in the dedicated RCT of the NECESSITY consortium.

Item Type:	Article
Keywords:	primary Sjögren’s syndrome, outcome measure, composite outcome, clinical trial
Date Deposited:	21 Jun 2022 00:45
Last Modified:	21 Jun 2022 00:45
URI:	https://oak.novartis.com/id/eprint/47282