Idiopathic Inflammatory Myopathies: Improving Diagnosis and Predicting Outcomes

特发性炎症性肌病：改善诊断和预测结果

• 批准号: 7022501
• 负责人: LISA CHRISTOPHER-STINE
• 金额: $ 13.47万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-05-05 至 2011-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7022501
• 关键词: biopsy; classification; clinical research; diagnosis; genetics; information systems; muscle; muscular dystrophy; phenotype

DESCRIPTION (provided by applicant): This Mentored Patient-Oriented Research Career Development Award will enable Dr. Christopher-Stine to acquire the vital methodologic skills and expertise to become an independent clinical investigator, focusing on the idiopathic inflammatory myopathies (IIM). The PI will combine her growing research experience and continued training in public health with the mentorship of Drs. Allan Gelber, Antony Rosen, and Paul Plotz. IIM, including dermatomyositis, polymyositis, and inclusion body myositis, constitute the largest subset of acquired myopathies and often affect adults in their prime. A major limitation of prior studies is the poor specificity of the diagnostic criteria used to classify IIM. The therapeutic implication for such misclassified patients is substantial. Thus, it is crucial to sharpen conventional classification criteria so that therapy is used appropriately. The published literature in IIM is also characterized by few formal prospective myositis cohort studies, yielding sparse evidence by which to reliably predict meaningful outcomes. To address and to overcome these important methodologic limitations in IIM, Dr. Christopher-Stine will interrogate the valuable NIH Protocol entitled "Studies in Natural History and Pathogenesis of Polymyositis and Dermatomyositis and Related Diseases." An Associate Investigator to this protocol, Dr. Christopher-Stine has participated in patient evaluations and is currently formally organizing the demographic, clinical, serologic, and radiographic data collected on 672 patients enrolled in the protocol since 1991. To date, no formal epidemiologic database has been established using this resource. This application proposes to access, organize and establish this protocol into a systematic relational database which will be utilized to address the following Specific Aims: (1) To determine the sensitivity and specificity of Magnetic Resonance Imaging (MRI) to discriminate among IIM, muscular dystrophy, and normal muscle; (2) To determine the diagnostic value of novel genetic expression profiling among patients presenting with a myositis phenotype who remain undiagnosed after utilizing conventional diagnostic classification schema; (3) To define longitudinal predictors of mortality, disability, and malignancy. With support from this award, the invaluable complementary mentorship skills, and the expertise of the listed collaborators, Dr. Christopher-Stine is poised to develop and launch her independent clinical research career.

描述（由申请人提供）：这项受过指导的以患者为导向的研究职业发展奖将使克里斯托弗·斯汀（Christopher-Stine）博士获得重要的方法论技巧和专业知识，成为独立的临床研究者，重点关注特发性炎症性肌病（IIM）。 PI将结合她不断增长的研究经验，并继续在公共卫生方面的培训和DR的指导。艾伦·盖尔伯（Allan Gelber），安东尼·罗森（Antony Rosen）和保罗·普洛茨（Paul Plotz）。 IIM，包括皮肌炎，多聚肌炎和包容体肌炎，构成了获得性肌病的最大部分，并且经常会影响成年人的质量。先前研究的主要局限性是用于对IIM进行分类的诊断标准的特异性差。对这种错误分类的患者的治疗意义是很大的。因此，这对于培养常规分类标准至关重要，因此可以适当地使用治疗。 IIM中发表的文献也以很少的正式前瞻性肌炎队列研究为特征，从而产生了稀疏的证据来可靠地预测有意义的结果。为了解决和克服IIM中这些重要的方法论局限性，克里斯托弗·斯泰恩（Christopher-Stine）博士将询问题为“自然史和多肌炎和皮肤病及相关疾病的自然史和发病机理研究的有价值的NIH方案”。克里斯托弗·斯汀（Christopher-Stine）博士是该方案的副研究人员，已经参与了患者评估，目前正正式组织了自1991年以来在该方案中招募的672名患者收集的人口，临床，血清学和射线照相数据。迄今为止，没有使用此资源建立正式的流行病学数据库。该应用程序建议在系统的关系数据库中访问，组织和建立该方案，该数据库将用于解决以下特定目的：（1）确定磁共振成像（MRI）的灵敏度和特异性（MRI）以区分IIM，肌肉性营养不良和正常肌肉； （2）确定出现肌炎表型的患者中新型遗传表达分析的诊断价值，这些患者在使用常规诊断分类模式后仍未诊断； （3）定义死亡率，残疾和恶性肿瘤的纵向预测指标。在该奖项的支持下，宝贵的补充指导技巧以及上市合作者的专业知识，克里斯托弗·斯汀（Christopher-Stine）博士准备开发和启动她独立的临床研究职业。