Developing Clinical Risk Predictions Rules in Early Diffuse Schleroderma

制定早期弥漫性硬皮病的临床风险预测规则

基本信息

批准号：
8209055
负责人：
Robyn Therese Domsic
金额：
$ 12.33万
依托单位：
UNIVERSITY OF PITTSBURGH AT PITTSBURGH
依托单位国家：
美国
项目类别：
财政年份：
2009
资助国家：
美国
起止时间：
2009-09-01 至 2014-12-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8209055
关键词：
Address Affect Angiotensin-Converting Enzyme Inhibitors Autoantibodies Autoimmune Diseases Biological Markers Blood Vessels Calibration Caring Classification Clinical Clinical Research Clinical Trials Clinical Trials Design Conflict (Psychology)Data Databases Derivation procedure Development Diffuse Diffuse Scleroderma Discrimination Disease Fibrosis Foundations Genetic Markers Goals Guidelines Hospitals Individual Inflammation Kidney Knowledge Laboratories Literature Medicine Mentors Methods Modeling Organ Outcome Outcome Measure Patient Care Management Patients Physicians Population Principal Investigator Prognostic Factor Publishing Rare Diseases Recording of previous events Regression Analysis Reporting Research Research Training Resources Rheumatism Risk Risk Factors Sample Size Scientist Scleroderma Serological Skin Symptoms Testing Time Training Training Support Trees Universities Validation Work arm base clinical application clinical practice cohort cytokine design experience high risk improved interest meetings mortality multidisciplinary patient population prognostic programs

项目摘要

DESCRIPTION (provided by applicant): Scleroderma is a multisystem autoimmune disease with the highest case specific mortality rate of the rheumatic diseases. Literature on scleroderma mortality and its associated predictors has been disparate. The reasons for this variability include the rarity of the disease, small sample sizes and different methodologic approaches. As such, there is no validated method for predicting short or long term mortality in scleroderma. The objective of this proposal is to develop both a two- and five-year mortality risk prediction rule for patients with early diffuse scleroderma while using state of the art methodologic guidelines and a true inception cohort from a large, well- characterized, prospectively followed scleroderma population at the University of Pittsburgh. Specific Aim la develops the prediction rules using regression analysis augmented by classification tree analysis. Specific Aim lb internally validates the rules using the University of Pittsburgh Scleroderma cohort. Specific Aim 2a externally validates the two-year mortality prediction rule using cohorts from the Royal Free Hospital and the Scleroderma Clinical Trials Consortium. Specific aim 2b externally validates the five-year mortality prediction rule using the Royal Free Hospital scleroderma cohort. The development of prediction rules to calculate an individual patient's risk of two- and five-year mortality and ascertain factors associated with survival will have direct research and clinical applications. These prediction rules will be useful in clinical practice and to identify individuals at high risk of short and longer term mortality for inclusion in clinical trials of new therapies for scleroderma when mortality is an outcome measure. The ultimate goal of this line of research is to develop prediction rules that will fulfill the promise of personalized medicine for patients with scleroderma. The experience of developing and vaUdating prediction rules, combined with advanced methodologic training through didactic coursework and directed tutorials, will provide invaluable clinical research training, and help me attain my goal of becoming an independent physician scientist dedicated to improving the care and outcomes of scleroderma patients.

描述（由申请人提供）：硬皮病是一种多系统自身免疫性疾病，具有风湿病的特定病例死亡率最高。关于硬皮病死亡率及其相关预测因子的文献是不同的。这种可变性的原因包括疾病的稀有性，小样本量和不同的方法论方法。因此，没有验证的方法可以预测硬皮病中短期或长期死亡率。该提案的目的是为早期弥漫性硬皮病患者制定两年和五年的死亡风险预测规则，同时使用艺术方法学指南，并从一个大型，良好的特征性，前瞻性地跟随匹兹堡大学的硬化症人群中的真实成立群体。特定目标使用分类树分析增强的回归分析来制定预测规则。特定的AIM LB在内部使用匹兹堡大学的硬皮病学院验证了规则。特定目标2a外部使用皇家自由医院和硬皮病临床试验联盟的同伙进行了两年的死亡率预测规则。特定的目标2B外部使用皇家自由医院硬皮病队列验证了五年的死亡率预测规则。预测规则的制定是计算单个患者患有五年和五年死亡率的风险以及与生存相关的确定因素的风险，将具有直接的研究和临床应用。这些预测规则将在临床实践中很有用，并确定在死亡率是一种结局指标时，在临床疗法中具有高度和长期死亡率的高风险。这项研究线的最终目标是制定预测规则，这些规则将符合硬皮病患者的个性化医学承诺。通过教学课程和指导教程，制定和vauding预测规则的经验，以及高级方法论培训，将提供宝贵的临床研究培训，并帮助我实现我成为一名独立的医师科学家的目标，致力于改善硬皮病患者的护理和成果。