Systemic Sclerosis-Associated Interstitial Lung Disease Response Index

项目摘要/摘要 系统性硬化症相关间质性肺疾病(SSC-ILD)影响大约80%的患者， 导致压倒性的发病率，是SSC的主要死亡原因之一。显然需要一种 系统开发的SSC-ILD反应指数，包括生理、放射和患者报告 结果衡量标准。根据风湿学(OMERACT)的结果衡量标准，研究人员 已获得关于制定南南合作--ILD反应指数的最终领域的认可。 首席调查员(PI)现在提出一种数据驱动方法，将核心项目集验证为 SSC-ILD的响应指数。我们的中心假设，制定并基于调查人员的专业知识 我们的初步数据是，可以使用一组核心变量来开发稳健的特定于SSC-ILD的 涵盖所有相关结果的反应指数，包括生理、放射和患者- 报告的措施。私人投资委员会将通过追求以下两个具体目标来检验这一假设： 1.进行前瞻性纵向观察性临床研究，以确定可靠、有效和有效的 以最近达成的共识为基础的一套综合反应指数的SSC-ILD措施 锻炼身体，以及 2.使用前瞻性的、数据驱动的、建立共识的技术来开发和量化评估 SSC-ILD反应指数的候选定义。这项建议利用了国际和平研究所的专业知识，以及 风湿病临床研究专家，肺部内科和放射学专家担任 共同调查员。此外，他还召集了著名的国际研究人员作为指导 委员会成员。

Project Summary/Abstract Systemic sclerosis-associated interstitial lung disease (SSc-ILD) affects approximately 80% of patients, induces overwhelming morbidity and is one of the leading causes of death in SSc. There is a distinct need for a systematically developed SSc-ILD response index that captures physiologic, radiologic, and patient-reported outcome measures. Under the Outcome Measures in Rheumatology (OMERACT) aegis, the investigators have received an endorsement on final domains for development of a SSc-ILD response index. The Principal Investigator (PI) now proposes a data-driven approach to validate that core set of items into a response index for SSc-ILD. Our central hypothesis, formulated and based on the expertise of the investigators and our preliminary data, is that a core set of variables can be used to develop a robust SSc-ILD-specific response index that spans the full range of relevant outcomes, including physiologic, radiographic and patient- reported measures. The PI will test the hypothesis by pursuing the following two specific aims: 1. Perform a prospective longitudinal observational clinical study to define a reliable, valid and responsive set of SSc-ILD measures for a combined response index, based on a recently completed consensus exercise, and 2. Employ prospective, data-driven, consensus-building techniques to develop and quantitatively evaluate candidate definitions for a response index for SSc-ILD. This proposal utilizes the expertise of the PI, an established clinical investigator in rheumatology, with experts in pulmonary medicine and radiology as co-investigators. In addition, he has gathered distinguished international researchers as the steering committee members.