University of Michigan, Ann Arbor.
University of Florence, Florence, Italy.
Arthritis Rheumatol. 2020 Jul;72(7):1049-1058. doi: 10.1002/art.41246. Epub 2020 May 18.
Systemic sclerosis (SSc) is an autoimmune rheumatic disease with heterogeneous clinical manifestations and a variable course in which the severity of the pathology dictates the disease prognosis and course. Among autoimmune rheumatic diseases, SSc has the highest mortality rate among all rheumatic diseases, though there are exciting new therapeutic targets that appear to halt the progression of SSc manifestations such as skin or lung fibrosis. In selected patients, high-intensity regimens with autologous stem cell transplantation can favorably modify the course. In what was once thought to be an untreatable disease, targeted therapies have now changed the outlook of SSc to a treatable disorder. Herein, we discuss the targeted therapies modifying the outlook on selected organ involvement and creating opportunities for future treatment. We also present a framework for defining low disease activity in SSc.
系统性硬化症(SSc)是一种自身免疫性风湿病,具有异质性的临床表现和多变的病程,其中病理学的严重程度决定了疾病的预后和病程。在自身免疫性风湿病中,SSc 的死亡率在所有风湿病中最高,尽管有一些令人兴奋的新的治疗靶点似乎可以阻止 SSc 表现如皮肤或肺纤维化的进展。在一些特定的患者中,高强度的自体干细胞移植方案可以有利地改变病程。在曾经被认为是无法治疗的疾病中,靶向治疗现在已经改变了 SSc 的治疗前景,使之成为一种可治疗的疾病。在这里,我们讨论了靶向治疗如何改变对选定器官受累的看法,并为未来的治疗创造机会。我们还提出了一个定义 SSc 低疾病活动度的框架。