Nelson Nathaniel C, Kogan Rebecca, Condos Rany, Hena Kerry M
Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Medicine, New York University, 301 E 17th St Suite 550, New York, NY 10003, USA.
J Clin Med. 2023 Dec 19;13(1):15. doi: 10.3390/jcm13010015.
Sarcoidosis is a systemic disease with heterogenous clinical phenotypes characterized by non-necrotizing granuloma formation in affected organs. Most disease either remits spontaneously or responds to corticosteroids and second-line disease-modifying therapies. These medications are associated with numerous toxicities that can significantly impact patient quality-of-life and often limit their long-term use. Additionally, a minority of patients experience chronic, progressive disease that proves refractory to standard treatments. To date, there are limited data to guide the selection of alternative third-line medications for these patients. This review will outline the pathobiological rationale behind current and emerging therapeutic agents for refractory or drug-intolerant sarcoidosis and summarize the existing clinical evidence in support of their use.
结节病是一种具有异质性临床表型的全身性疾病,其特征是在受影响的器官中形成非坏死性肉芽肿。大多数疾病要么自发缓解,要么对皮质类固醇和二线疾病改良疗法有反应。这些药物具有多种毒性,可显著影响患者的生活质量,并常常限制其长期使用。此外,少数患者会经历慢性进行性疾病,对标准治疗无效。迄今为止,指导为这些患者选择替代性三线药物的数据有限。本综述将概述难治性或不耐受药物的结节病的现有和新兴治疗药物背后的病理生物学原理,并总结支持其使用的现有临床证据。