Systemic Sclerosis-Associated Interstitial Lung Disease Response Index

项目总结/摘要 系统性硬化症相关间质性肺病（SSc-ILD）影响约80%的患者， 引起压倒性的发病率，是SSc死亡的主要原因之一。有一个明显的需要， 系统开发的SSc-ILD缓解指数，可捕获生理学、放射学和患者报告的 结果测量。在流变学结果测量（OMERACT）的支持下， 已获得关于开发SSc-ILD缓解指数的最终领域的认可。 主要研究者（PI）现在提出了一种数据驱动的方法，以验证该核心项目集， SSc-ILD的缓解指数。我们的中心假设，制定并基于调查人员的专业知识， 和我们的初步数据，是一组核心变量可以用来开发一个强大的SSc-ILD特异性 反应指数涵盖了所有相关结局，包括生理学、放射学和患者- 报告的措施。PI将通过追求以下两个具体目标来检验假设： 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 基于最近完成的共识，一组SSc-ILD指标用于组合缓解指数 锻炼和 2.采用前瞻性的、数据驱动的、建立共识的技术来开发和定量评估 用于SSc-ILD的响应指数的候选定义。该提案利用PI的专业知识， 在风湿病学方面建立了临床研究者，与肺部医学和放射学专家一起， 共同调查员此外，他还聚集了杰出的国际研究人员， 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.