Systemic sclerosis (SSc) is a rare, systemic autoimmune disease of unknown etiology. Among the systemic rheumatic diseases, SSc carries the highest mortality, in part due to the historical lack of disease modifying therapies. Recently, landmark randomized controlled trials (RCTs) have been conducted that have illustrated the heterogeneous nature of SSc and furthered our understanding of the key inflammatory and fibrotic pathways involved in SSc pathogenesis. Although SSc affects various organ systems, RCTs have focused on investigating treatments for diffuse cutaneous sclerosis (dcSSc) and interstitial lung disease (ILD). While recent RCTs for dcSSc have failed to demonstrate a treatment benefit, the outcomes of two RCTs led to the approval of two novel therapies for SSc-ILD: nintedanib and tocilizumab. This review summarizes the salient outcome data from recent SSc trials within a practical clinical framework and points out gaps in knowledge that may help inform the design of future SSc studies.

系统性硬化症 (SSc) 是一种罕见的、病因不明的系统性自身免疫性疾病。在系统性风湿性疾病中，SSc 的死亡率最高，部分原因是历史上缺乏疾病改善疗法。最近，已经进行了具有里程碑意义的随机对照试验 (RCT)，这些试验说明了 SSc 的异质性，并进一步加深了我们对参与 SSc 发病机制的关键炎症和纤维化途径的理解。尽管 SSc 影响各种器官系统，但 RCT 的重点是研究弥漫性皮肤硬化症 (dcSSc) 和间质性肺病 (ILD) 的治疗方法。虽然最近针对 dcSSc 的随机对照试验未能证明治疗益处，但两项随机对照试验的结果导致两种用于 SSc-ILD 的新疗法获得批准：尼达尼布和托珠单抗。