STAR aims to be ‘gold standard’ for outcome measures in Sjögren’s syndrome
Click Here to Manage Email Alerts
The Sjögren’s Tool for Assessing Response, or STAR, a candidate composite efficacy endpoint for clinical trials, includes all disease features and has demonstrated sensitivity to change, according to researchers.
“For decades, evidence-based therapy in primary Sjögren’s syndrome (pSS) has largely been based on sicca features or patient-reported outcomes (PROs),” Raphaele Seror, MD, PhD, of the Paris-Saclay University Center for Immunology of Viral Infections and Autoimmune Diseases, in France, and colleagues wrote in the Annals of the Rheumatic Diseases. “STAR aims to resolve the issues on current outcome measures in pSS and is intended for use in clinical trials as an efficacy endpoint.”
To develop the STAR system, Seror and colleagues followed a four-step process in gathering experts, developing options and cast their final votes. In the first step, investigators examined the main tenants responsible for identifying treatment response in primary Sjögren’s syndrome. Researchers used data from two trials investigating rituximab (Rituxan, Genentech), because findings from those studies demonstrated that the drug should work for some patients and endpoints, even if it was not effective in all patients, the authors wrote.
Seror and colleagues then identified responder subsets, which markers were sensitive to changes and selected items, and defined what patient response meant in the context of the evaluation. In all, the panel made up of 78 Sjogren’s experts, including 57 clinicians and 21 scientists, as well as 20 patients.
The research team determined drafts for what would become the options put forth for the panel. In addition to combining options determined in the previous step, panelists had the ability to make suggestions or alternate tools for measuring outcomes. The researchers selected designs that received 50% or more of the vote, the authors wrote. Finally, the authors evaluated each candidate’s sensitivity to change and selected the most appropriate system.
Deliberations to land on methods to be included in the new evaluation criteria were derived from nine randomized control trials and various consensus methods. Trials were evaluated based panel votes. In all, the panel identified five core areas and 227 options to be tested for sensitivity to change. A meta-analysis of the remaining 20 options was conducted, and the final candidate STAR was selected based on a vote considering “metrological properties and clinical relevance,” the authors wrote. The authors aimed to validate the system by the NECESSITY consortium in a dedicated controlled trial.
The panelists chose “the published minimal clinically important difference (MCID) as the response cut-off for clinESSDAI (3 points) and ESSPRI (1 point),” and rejected a “no worsening” clause from the final STAR, they wrote.
“Even though this process relied on a nearly never-equal number of experts and RCTs, further to the present retrospective validation, STAR has to be prospectively validated in an independent population in the NECESSITY RCT,” Seror and colleagues wrote. “The strength of this validation step is its evaluation of the psychometric properties of STAR, in particular its discriminant capacity in an interventional study where active and placebo arms will be compared.”
The authors recommend the use of STAR to evaluate diverse patient populations to validate it as a “gold standard outcome measure,” in patients with primary Sjögren’s syndrome.
Collapse
Read more about
Click Here to Manage Email Alerts