Provisional Combined Response Index For Patients With Diffuse Systemic Sclerosis

弥漫性系统性硬化症患者的临时综合缓解指数

• 批准号: 7485930
• 负责人: Daniel Eric Furst
• 金额: $ 14.49万
• 依托单位: UNIVERSITY OF CALIFORNIA LOS ANGELES
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-08-01 至 2010-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7485930
• 关键词: American; Area Under Curve; Arthritis; Characteristics; Clinical; Clinical Research; Clinical Trials; Clinical Trials Design; Consensus; Cutaneous; Data; Data Set; Development; Diffuse; Diffuse Scleroderma; Disease; Exercise; Face; Funding; Goals; Gold; Heterogeneity; Individual; International; Language; Lead; Measurement; Measures; Meta-Analysis; Methodology; Morbidity - disease rate; Natural History; Numbers; Observational Study; Organ; Outcome; Outcome Measure; Paper; Pathogenesis; Patient Outcomes Assessments; Patients; Principal Investigator; Rate; Receiver Operating Characteristics; Research; Research Personnel; Rheumatism; Rheumatoid Arthritis; Rheumatology; Scleroderma; Score; Sensitivity and Specificity; Skin; Specific qualifier value; Standards of Weights and Measures; Structure; Systemic Scleroderma; Techniques; Testing; Therapeutic Agents; Time; Treatment Efficacy; Validation; Validity and Reliability; Work; base; college; design; drug development; improved; indexing; interest; mortality; patient oriented; programs; prospective; response; sound

Systemic sclerosis (Scleroderma, SSc) can be associated with a high morbidity and mortality. The complexity and heterogeneity of the disease mandates a composite response index that will capture different organ involvement and patient-reported outcomes. There is a critical need for an SSc-combined response index (SSc-CRI) to to facilitate drug development and to allow comparison among clinical trials in SSc, as a similar index did in rheumatoid arthritis. We have assembled a group of international experts who have have expertise in clinical trial design and development of response criteria in different arthritides. UCLA Clinical Coordinating Center has successfully coordinated recently concluded NIH-funded SSc clinical trials. We took the first step in the developing an SSc-CRI by conducting a structured Delphi exercise to develop a provisional core set of items for clinical trials. The Delphi exercise identified 11 domains relevant to diffuse SSc clinical trials. Our long-term goals are to improve methodologically sound SSc clinical trials and increase interest in drug development for SSc. This application aims to develop an SSc-CRI to be used as an outcome measure in diffuse SSc clinical trials. Our central hypothesis is that a core set of variables for patients with diffuse SSc can be used to develop a formula for an SSc-CRI with robust test characteristics. Our Specific Aims for the 3-year proposal are: Specific Aim1. Perform a prospective longitudinal observational clinical study in 200 patients with diffuse SSc to define a reliable, valid and responsive set of SSc measures for an SSc-CRI, based on a recently completed Delphi exercise. Specific Aim 2. Employ a prospective, data-driven, consensus building techniques to develop and quantitatively evaluate candidate definitions for an SSc-CRI for diffuse SSc. The research proposed in this application is significant because it is expected to provide a single SSc-CRI for diffuse SSc, thereby providing an impetus for drug development and improved assessment of efficacy of therapeutic agents in clinical trials. Lay Language: We propose to develop a response criteria for clinical trials in diffuse scleroderma, that can facilitate drug development and hopefully, can lead to effective treatments for scleroderma.

系统性硬化症（硬皮病，SSc）可能与高发病率和死亡率相关。的 疾病的复杂性和异质性要求采用综合反应指数， 器官受累和患者报告的结果。迫切需要SSc联合应对措施 指数（SSc-CRI），以促进药物开发，并允许在SSc的临床试验之间进行比较，作为一个 类风湿性关节炎也有类似的指数。我们召集了一组国际专家， 在不同关节炎的临床试验设计和反应标准开发方面的专业知识。UCLA临床 协调中心已成功协调最近结束的NIH资助的SSc临床试验。我们带 通过进行结构化的德尔菲练习， 临床试验的暂定核心项目集。德尔菲研究确定了11个与扩散相关的领域， SSc临床试验。我们的长期目标是改进方法学上合理的SSc临床试验， 对SSc药物开发感兴趣。本申请旨在开发一种SSc-CRI， 弥漫性SSc临床试验的结果测量。我们的中心假设是， 具有弥漫性SSc的患者可用于开发具有稳健测试特征的SSc-CRI的配方。 我们三年计划的具体目标是： 具体目标1。在200例弥漫性SSc患者中进行前瞻性纵向观察性临床研究 为SSc-CRI定义一套可靠、有效和响应迅速的SSc措施， 完成了德尔菲练习。 具体目标2。采用前瞻性的、数据驱动的、建立共识的技术来开发和 定量评估弥漫性SSc的SSc-CRI的候选定义。 本申请中提出的研究是重要的，因为它有望提供单个SSc-CRI 对于弥漫性SSc，从而为药物开发和改善对药物疗效的评估提供动力。 临床试验中的治疗药物。 我们建议为弥漫性硬皮病的临床试验制定一个反应标准， 促进药物开发，并有望导致硬皮病的有效治疗。