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系统性硬化症的可治疗特征

Treatable Traits in Systemic Sclerosis.

作者信息

Amati Francesco, Bongiovanni Gabriele, Tonutti Antonio, Motta Francesca, Stainer Anna, Mangiameli Giuseppe, Aliberti Stefano, Selmi Carlo, De Santis Maria

机构信息

Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, Milan, Italy.

Respiratory Unit, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy.

出版信息

Clin Rev Allergy Immunol. 2023 Oct;65(2):251-276. doi: 10.1007/s12016-023-08969-x. Epub 2023 Aug 21.

Abstract

Systemic sclerosis (SSc) is a chronic systemic disease within the spectrum of connective tissue diseases, specifically characterized by vascular abnormalities and inflammatory and fibrotic involvement of the skin and internal organs resulting in high morbidity and mortality. The clinical phenotype of SSc is heterogeneous, and serum autoantibodies together with the extent of skin involvement have a predictive value in the risk stratification. Current recommendations include an organ-based management according to the predominant involvement with only limited individual factors included in the treatment algorithm. Similar to what has been proposed for other chronic diseases, we hypothesize that a "treatable trait" approach based on relevant phenotypes and endotypes could address the unmet needs in SSc stratification and treatment to maximize the outcomes. We provide herein a comprehensive review and a critical discussion of the literature regarding potential treatable traits in SSc, focusing on established and candidate biomarkers, with the purpose of setting the bases for a precision medicine-based approach. The discussion, structured based on the organ involvement, allows to conjugate the pathogenetic mechanisms of tissue injury with the proposed predictors, particularly autoantibodies and other serum biomarkers. Ultimately, we are convinced that precision medicine is the ideal guide to manage a complex condition such as SSc for which available treatments are largely unsatisfactory.

摘要

系统性硬化症(SSc)是一种结缔组织疾病谱中的慢性全身性疾病,其具体特征为血管异常以及皮肤和内脏器官的炎症和纤维化,导致高发病率和死亡率。SSc的临床表型具有异质性,血清自身抗体以及皮肤受累程度在风险分层中具有预测价值。目前的建议包括根据主要受累器官进行基于器官的管理,治疗算法中仅纳入有限的个体因素。与针对其他慢性疾病所提出的建议类似,我们假设基于相关表型和内型的“可治疗特征”方法可以满足SSc分层和治疗中未满足的需求,以实现最佳治疗效果。我们在此对有关SSc潜在可治疗特征的文献进行全面综述和批判性讨论,重点关注已确定的和候选生物标志物,目的是为基于精准医学的方法奠定基础。基于器官受累情况进行结构化的讨论,能够将组织损伤的发病机制与所提出的预测指标(特别是自身抗体和其他血清生物标志物)结合起来。最终,我们坚信精准医学是管理像SSc这样复杂疾病的理想指南,因为目前可用的治疗方法大多不尽人意。

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