Disease Modification and Other Trials in Systemic Sclerosis Have Come a Long Way, But Have to Go Further

摘要

In this issue of Arthritis Care & Research, the View article by Mendoza et al about clinical trial design in systemic sclerosis (SSc; scleroderma) suggests that there cannot be a universal design for clinical trials of disease modification in SSc because the disease has so many different presentations (1). However, it is still important in any randomized controlled trial (RCT) to use proper statistics, including sample size calculations, to be able to recruit subjects (feasible), and ideally to have subjects enrolled that are similar to SSc patients with comparable organ-based disease activity (generalizable). There will not be a single design for every trial, but basic principles must be adhered to. We have written this article as a counterview to the views by Mendoza et al (1), but most of the opinions are in agreement. The headings are ordered in a way that would mimic the process of clinical trial design and conduct (general principles, end points, and discussion of organ-specific trials in SSc skin and lung). Examples from trials of various rheumatic diseases have been provided to illustrate various aspects of trial design.