News and failures from recent treatment trials in systemic sclerosis.

Abstract

There have been many recent trials in systemic sclerosis (SSc) that have explored treatment for skin or lung. Some have been encouraging, but there has also been disappointment reflecting potential limitations of treatment effect of study design. These trials are discussed and reviewed. Studies conducted in SSc are described and discussed with a focus on endpoint selection and trial design as well as potential mechanism of action and treatment effect. Studies have included very encouraging trials of interleukin 6 blockade, immunosuppression, and broad-spectrum tyrosine kinase inhibition. Other trials including recent studies of peroxisome proliferator-activated receptor agonists and specific intracellular signaling inhibitors such as imatinib or anti-transforming growth factor beta blocking strategies have been more disappointing. Trial design is improving, and overall, there are now almost positive trials using agents with great promise, and studies are also providing important biological insight into SSc. It is hoped that ongoing studies will further progress the field and move it toward better treatments for SSc that still represent a major unmet medical need.