Ligon Colin, Hummers Laura K
Division of Rheumatology, Department of Medicine, Johns Hopkins University School of Medicine, 5200 Eastern Avenue, Mason F. Lord Building, Center Tower, Suite 4100, Baltimore, MD, 21224, USA.
Curr Rheumatol Rep. 2016 Mar;18(3):17. doi: 10.1007/s11926-016-0565-0.
Scleroderma is a heterogenous disease characterized by autoimmunity, a characteristic vasculopathy, and often widely varying extents of deep organ fibrosis. Recent advances in the understanding of scleroderma's evolution have improved the ability to identify subgroups of patients with similar prognosis in order to improve risk stratification, enrich clinical trials for patients likely to benefit from specific therapies, and identify promising therapeutic targets for intervention. High-throughput technologies have recently identified fibrotic and inflammatory effectors in scleroderma that exhibit strong prognostic ability and may be tied to disease evolution. Increasingly, the use of collections of assayed circulating proteins and patterns of gene expression in tissue has replaced single-marker investigations in understanding the evolution of scleroderma and in objectively characterizing disease extent. Lastly, identification of shared patterns of disease evolution has allowed classification of patients into latent disease subtypes, which may allow rapid clinical prognostication and targeted management in both clinical and research settings. The concept of biomarkers in scleroderma is expanding to include nontraditional measures of aggregate protein signatures and disease evolution. This review examines the recent advances in biomarkers with a focus on those approaches poised to guide prospective management or themselves serve as quantitative surrogate disease outcomes.
硬皮病是一种异质性疾病，其特征为自身免疫、特征性血管病变，且深部器官纤维化程度往往差异很大。在硬皮病演变过程的理解方面取得的最新进展，提高了识别预后相似患者亚组的能力，以便改善风险分层、丰富可能从特定疗法中获益患者的临床试验，并确定有前景的干预治疗靶点。高通量技术最近在硬皮病中识别出了具有强大预后能力且可能与疾病演变相关的纤维化和炎症效应因子。越来越多地，在理解硬皮病演变和客观表征疾病程度方面，使用检测循环蛋白集合和组织中基因表达模式已取代了单标记物研究。最后，疾病演变共同模式的识别已使患者能够被分类为潜在疾病亚型，这可能有助于在临床和研究环境中进行快速临床预后评估和靶向管理。硬皮病中生物标志物的概念正在扩展，以纳入蛋白质聚集特征和疾病演变的非传统测量方法。本综述探讨了生物标志物的最新进展，重点关注那些有望指导前瞻性管理或本身可作为定量替代疾病结局的方法。
