系统性硬化症的病原学和组织病理学方面、流行病学、分类系统和疾病结果测量的叙述性综述。

IF 8.4 2区 医学 Q1 ALLERGY
Clinical Reviews in Allergy & Immunology Pub Date : 2023-06-01 Epub Date: 2022-03-07 DOI:10.1007/s12016-022-08929-x
Maria-Grazia Lazzaroni, Silvia Piantoni, Fabrizio Angeli, Stefania Bertocchi, Franco Franceschini, Paolo Airò
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引用次数: 0

摘要

系统性硬化症(SSc)是一种罕见的系统性自身免疫性疾病,其特点是存在三个主要角色:血管病变、免疫激活和纤维化。这一病理过程转化为临床表现,不同患者在受累器官类型、疾病严重程度和预后方面存在很大差异。这种异质性是 SSc 的一个主要特征,再加上疾病早期症状轻微,这就解释了为什么在建立分类标准、定义患者亚群和疾病预后方面存在很大困难。在临床试验中,疾病结果的定义尤为重要,因为临床试验的目的是提供可靠的终点,以衡量某种药物或干预措施的疗效大小。因此,在过去的几年里,人们越来越努力地设计疾病活动、损害、严重程度和对治疗的反应的测量方法,通常采用综合指数的形式。在考虑疾病结果时,患者的经历是一个相关的补充方面。在过去几年中,临床实践和临床试验中越来越多地使用能够捕捉这种体验的工具,即患者报告的结果,并将其作为主要和次要终点。因此,这篇关于 SSc 的综合叙述性综述将涵盖病原学和组织病理学方面、流行病学、分类系统和疾病结果测量,以便重点关注与临床研究和临床试验设计相关的问题。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A Narrative Review of Pathogenetic and Histopathologic Aspects, Epidemiology, Classification Systems, and Disease Outcome Measures in Systemic Sclerosis.

A Narrative Review of Pathogenetic and Histopathologic Aspects, Epidemiology, Classification Systems, and Disease Outcome Measures in Systemic Sclerosis.

Systemic sclerosis (SSc) is a rare systemic autoimmune disease, characterized by the presence of three main actors: vasculopathy, immune activation, and fibrosis. This pathologic process is then translated in a clinical picture with great variability among different patients in terms of type of organ involvement, disease severity and prognosis. This heterogeneity is a main feature of SSc, which, in addition to the presence of early phases of the disease characterized by mild symptoms, can explain the high difficulty in establishing classification criteria, and in defining patients' subsets and disease outcomes. The definition of disease outcomes is particularly relevant in the setting of clinical trials, where the aim is to provide reliable endpoints, able to measure the magnitude of the efficacy of a certain drug or intervention. For this reason, in the last years, increasing efforts have been done to design measures of disease activity, damage, severity, and response to treatment, often in the context of composite indexes. When considering disease outcomes, the experience of the patient represents a relevant and complementary aspect. The tools able to capture this experience, the patient-reported outcomes, have been increasingly used in the last years in clinical practice and in clinical trials, both as primary and secondary endpoints. This comprehensive narrative review on SSc will therefore cover pathogenetic and histopathologic aspects, epidemiology, classification systems, and disease outcome measures, in order to focus on issues that are relevant for clinical research and design of clinical trials.

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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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