Department of Rheumatology, University Hospital Basel , Basel, Switzerland.
Expert Rev Clin Pharmacol. 2020 Nov;13(11):1203-1218. doi: 10.1080/17512433.2020.1832466. Epub 2020 Oct 19.
Systemic sclerosis (SSc) is a rare, difficult to treat disease with profound effects on quality of life and high mortality. Complex and incompletely understood pathophysiologic processes and greatly heterogeneous clinical presentations and outcomes have hampered drug development.
This review summarizes the currently available immunosuppressive and antifibrotic therapies and discusses novel approaches for the treatment of SSc. We reviewed the literature using the MEDLINE and ClinicalTrial.gov databases between May and September 2020.
Available immunosuppressive and antifibrotic drugs only modestly impact the course of the disease. Most drugs are currently only investigated in the subset of patients with early diffuse cutaneous SSc. In this patient population, hematopoietic stem-cell transplantation is currently the only treatment that has demonstrated reversal of lung involvement, enhanced quality of life and reduced long-term mortality, but carries the risk of short-term treatment-related mortality. A great need to provide better therapeutic options to patients exists also for those patients who have limited cutaneous skin involvement. A better understanding of SSc pathophysiology has enabled the identification of numerous new therapeutic targets. The progress made in the design of clinical trials and outcome parameters will likely result in the improvement of effective management options.
系统性硬化症（SSc）是一种罕见的、难以治疗的疾病，对生活质量有深远影响，死亡率高。复杂且不完全了解的病理生理过程以及临床表现和结果的高度异质性，阻碍了药物的开发。
本文综述了目前可用的免疫抑制剂和抗纤维化治疗方法，并讨论了治疗 SSc 的新方法。我们使用 MEDLINE 和 ClinicalTrials.gov 数据库，在 2020 年 5 月至 9 月期间对文献进行了综述。
目前可用的免疫抑制剂和抗纤维化药物仅能适度影响疾病的进程。大多数药物目前仅在早期弥漫性皮肤型 SSc 的患者亚组中进行研究。在这一患者群体中，造血干细胞移植是目前唯一显示出逆转肺受累、提高生活质量和降低长期死亡率的治疗方法，但有短期治疗相关死亡率的风险。对于那些皮肤受累有限的患者，也存在着为他们提供更好的治疗选择的巨大需求。对 SSc 病理生理学的深入了解使人们能够确定许多新的治疗靶点。临床试验和结果参数设计方面的进展，有望改善有效的治疗选择。
