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 skinor lung. Some have been encouraging, but there has also been disappointment reflecting potentiallimitations of treatment effect of study design. These trials are discussed and reviewed. Studies conductedin SSc are described and discussed with a focus on endpoint selection and trial design as wellas potential mechanism of action and treatment effect. Studies have included very encouraging trialsof interleukin 6 blockade, immunosuppression, and broad-spectrum tyrosine kinase inhibition. Othertrials including recent studies of peroxisome proliferator-activated receptor agonists and specificintracellular signaling inhibitors such as imatinib or anti-transforming growth factor beta blockingstrategies have been more disappointing. Trial design is improving, and overall, there are now almostpositive trials using agents with great promise, and studies are also providing important biologicalinsight into SSc. It is hoped that ongoing studies will further progress the field and move it towardbetter treatments for SSc that still represent a major unmet medical need.