Division of Rheumatology, Department of Medicine, University of Michigan, Ann Arbor, MI, USA.
Division of Pulmonary and Critical Care Medicine, David Geffen School of Medicine, University of California at Los Angeles, Los Angeles, CA, USA.
Rheumatology (Oxford). 2019 Apr 1;58(4):567-579. doi: 10.1093/rheumatology/key151.
SSc is a rare CTD that affects multiple organ systems, resulting in substantial morbidity and mortality. Evidence of interstitial lung disease (ILD) is seen in ∼80% of patients with SSc. Currently there is no approved disease-modifying treatment for ILD and few effective treatment options are available. CYC is included in treatment guidelines, but it has limited efficacy and is associated with toxicity. MMF is becoming the most commonly used medication in clinical practice in North America and the UK, but its use is not universal. Newer agents targeting the pathogenic mechanisms underlying SSc-ILD, including fibrotic and inflammatory pathways, lymphocytes, cell-cell and cell-extracellular membrane interactions, hold promise for better treatment outcomes, including improved lung function, patient-related outcomes and quality of life. Here we review ongoing trials of established and novel agents that are currently recruiting patients with SSc-ILD.
硬皮病（SSc）是一种罕见的 CTD，会影响多个器官系统，导致发病率和死亡率显著增加。约 80%的 SSc 患者存在间质性肺病（ILD）的证据。目前，ILD 尚无获批的疾病修正治疗药物，有效的治疗选择也很少。CYC 被纳入治疗指南，但疗效有限且与毒性相关。MMF 在北美和英国的临床实践中已成为最常用的药物，但并非普遍使用。针对 SSc-ILD 潜在发病机制的新型药物，包括纤维化和炎症途径、淋巴细胞、细胞-细胞和细胞-细胞外膜相互作用，为改善治疗结果（包括改善肺功能、患者相关结局和生活质量）带来了希望。在此，我们综述了正在招募 SSc-ILD 患者的现有和新型药物的临床试验。
