Systemic sclerosis trial design moving forward. Issue 2 (May 2016)

• Record Type: Journal Article
• Authors: Johnson, Sindhu R.; Khanna, Dinesh; Allanore, Yannick; Matucci-Cerinic, Marco; Furst, Daniel E.
• Is Part Of: Journal of scleroderma and related disorders. Volume 1:Issue 2(2016)
• Page Start: 177
• Page End: 180
• Publication Date: 2016-05
• Subjects: Bayesian -- Cross-over trial -- Enriched enrollment randomized withdrawal design -- Randomized controlled trials -- Randomized placebo phase design -- Scleroderma; Scleroderma (Disease) -- Periodicals; Systemic scleroderma -- Periodicals; Fibrosis -- Periodicals; 616.544
• DOI: 10.5301/jsrd.5000198
• Languages: English
• ISSNs: 2397-1983

Abstract

The 2013 American College of Rheumatology–European League Against Rheumatism (ACR–EULAR) classification criteria for systemic sclerosis (SSc) have shifted the construct of SSc. The new reality is that patients recruited for trials may not be so severe and not so advanced. We can now look for therapeutics that might stop disease evolution and/or prevent organ involvement. This article highlights recent advances in research methodology, and broadens the potential range of design and analytic considerations when planning an SSc trial.