Systemic Sclerosis-Associated Interstitial Lung Disease Response Index

系统性硬化症相关间质性肺疾病反应指数

• 批准号: 9761276
• 负责人: DINESH KHANNA
• 金额: $ 27.39万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-01 至 2021-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9761276
• 关键词: Affect; Area Under Curve; Assessment tool; Biometry; Cause of Death; Characteristics; Clinic; Clinical Investigator; Clinical Research; Clinical Trials; Clinical Trials Design; Committee Members; Communities; Connective Tissue Diseases; Consensus; Data; Development; Diagnostic radiologic examination; Diffuse Scleroderma; Exercise; Face; Funding; Gold; International; Interstitial Lung Diseases; Intervention; Knowledge; Left; Lung; Measures; Methodology; Michigan; Morbidity - disease rate; National Institute of Arthritis and Musculoskeletal and Skin Diseases; Observational Study; Outcome; Outcome Measure; Paper; Pathogenesis; Patient Outcomes Assessments; Patients; Pharmaceutical Preparations; Pharmacology; Physiological; Placebos; Principal Investigator; Pulmonary Fibrosis; Pulmonology; Radiology Specialty; Randomized Controlled Trials; Receiver Operating Characteristics; Reporting; Research; Research Personnel; Rheumatology; Scleroderma; Sensitivity and Specificity; Specific qualifier value; Structure; Systemic Scleroderma; Techniques; Testing; Time; Universities; base; idiopathic pulmonary fibrosis; indexing; interest; profiles in patients; prospective; recruit; response; working group

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.

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， 建立了风湿病学临床研究者，拥有肺部医学和放射学专家， 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。