Measuring Disease Activity and Outcomes in Clinical Trials

Systemic sclerosis (scleroderma, SSc) has seen a substantial progress in the development and validation of outcome measures. This chapter outlines and discusses the outcome measures that are feasible, reliable, and valid for randomized controlled trials (RCTs) in SSc. This chapter will first highlight the key characteristics of an outcome measure and will then present important outcome measures used in RCTs in SSc to evaluate skin involvement, but also visceral manifestations (notably including assessment of cardio-pulmonary manifestations or gastro-intestinal outcomes), musculoskeletal features, peripheral vascular disease, or quality of life. In this overview of SSc-assessment in RCTs, a specific insight will also be provided on combined response indices or activity indices, as well as patients-reported outcomes and other validated outcome measures.