Rheumatic Diseases Research Core Centers

系统性硬化症(SSC)是一种罕见的复杂风湿性疾病，累及多个器官系统，往往会导致致命的后果。它仍然是最难管理的风湿病之一，有效的治疗方法有限。在更快地了解SSc的发病机制和寻找新的治疗方法方面，有几个障碍。首先，SSC的相对稀缺性和这些患者对专业临床护理的需求，使得转化性研究集中在具有足够的患者基础和样本/组织收集计划的选定机构。因此，许多研究人员很少或根本无法接触到患者样本/组织。即使对于看到相对大量患者的研究人员来说，证明临床疾病和正在研究的病原学特征之间的关联的大样本规模也可能被证明是难以捉摸的。其次，患者表现和疾病进展的异质性导致了解发病机制的方法支离破碎，不同的研究人员研究不同的疾病表现，例如，皮肤纤维化与肺动脉高压的比较。第三，先进的技术，如微阵列和蛋白质组学，使用不同的平台，使得跨不同地点的研究很难解释。在为解决这些问题而成立的SSC研究人员联盟的授权下，这个核心中心将利用现有的机构资源形成四个核心，为先进技术提供患者样本和公共平台，旨在服务于联盟研究人员广泛的临床和翻译基础科学利益。行政核心(核心A)将为核心调查员提供结构、沟通和充实。两个核心(核心B和C)将分别排列来自SSC患者的皮肤和肺的病理标本，用于快速免疫组织化学评估目标蛋白。两个核心将为财团调查人员提供强大的蛋白质组(核心D)和微阵列(核心E)技术。所有核心将额外使用规模经济和核心资金，以正常成本的一小部分提供服务。此外，跨核心收集的公共平台和临床数据将为理解临床-病理关联提供一个大型、通用的数据库。

Systemic sclerosis (SSc) is a rare, complex rheumatic disease involving multiple organ systems with a frequently fatal outcome. It remains one of the most difficult rheumatic disease to manage, with limited effective therapies. There are several impediments to more rapid advances in understanding SSc pathogenesis and finding new treatments. First, the relative rarity of SSc and need of these patients for specialty clinical care has concentrated translational studies in select institutions having an adequate patient base and a program for sample/tissue collection. Thus many investigators have little or no access to patient samples/tissues. Even for investigators seeing relatively large numbers of patients, large sample sizes for proving associations between clinical disease and pathogenic features under study can prove elusive. Second, the heterogeneity of patient presentation and disease progression leads to fragmented approaches to understanding pathogenesis, different investigators studying different disease manifestations, for example, skin fibrosis compared to pulmonary arterial hypertension. Third, advanced technologies, such as microarray and proteomics are applied using different platforms, rendering interpretation of studies across different sites difficult. Empowered by a Consortia of SSc investigators formed to address these problems, this Core Center will leverage existing institutional resources to form four cores, providing patient samples and common platforms for advanced technologies designed to serve the broad clinical, translational basic scientific interests of Consortia Investigators. The Administrative Core (Core A), will provide structure, communication and enrichment of Core Investigators. Two cores (Cores B and C) will array pathological specimens from skin and lung, respectively, from SSc patients for rapid immunohistochemical evaluation of target proteins. Two cores will provide Consortia Investigators access to powerful Proteomic (Core D) and Microarray (Core E) technologies. All Cores will additionally use economy-of-scale and Core funding to provide services at a fraction of normal cost. In addition, the common platform and clinical data collected across cores will provide a large, common database for understanding clinical-pathological associations.