Abstract
The pathogenesis of systemic sclerosis (SSc) is complex and largely unclear. The clinical heterogeneity of the disease and its progression over a number of years makes the choice of endpoints in the design of clinical trials difficult. The overwhelming need in this disease is to diagnose it early and identify those patients who will benefit most from early, aggressive treatment that potentially can alter the clinical disease course. To achieve this, innumerable challenges must be overcome. This article reviews data from recent clinical trials and the lessons derived from retrospective observational studies, databases, and patient registries. Taken together, these observations will help to improve our understanding of the diverse clinical course of SSc and permit refinement of existing outcome measures for the design of future clinical trials, in which the likelihood of observing a positive treatment effect with the drugs at our disposal will be maximized.
Highlights
As is comprehensively reported in the previous reviews in this supplement, systemic sclerosis (SSc) is a multisystem disorder with a complex pathogenesis that remains to be elucidated
It is apparent that a combination of environmental or genetic factors leads to early immune activation and even earlier vascular damage, which leads through a variety of signals, to myofibroblast differentiation and proliferation
The results indicated that patients entered the studies with an mRSS in the region of 27, this reduced to about 13 at the 24-month point, representing a 50% spontaneous reduction over 2 years
Summary
As is comprehensively reported in the previous reviews in this supplement, systemic sclerosis (SSc) is a multisystem disorder with a complex pathogenesis that remains to be elucidated. Some observational studies initially appeared to demonstrate improved skin thickening, significant benefit in 5-year mortality and a decrease in development of visceral disease, these benefits were not confirmed in a well controlled, randomized, double-blind trial [5].
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
