Studies in Scleroderma and Cystic Fibrosis Osteoporosis

描述（由申请人提供）：为研究项目提供支持 在流变学领域，这将构成结构化指导的基础 计划为年轻的研究人员追求职业生涯在以病人为导向的临床 research.本申请有两个主要的具体研究目标：1）开发新的 用于临床试验的硬皮病皮肤评估的结果测量 和2）确定患者骨质疏松症的患病率和进展 囊性纤维化（CF）这两个项目将直接关系到 促进所研究人群的健康。临床研究 硬皮病，包括治疗试验，由于缺乏 疾病活动性的可靠和精确的结果测量。皮肤增厚 和纤维化是糖尿病患者发病和功能障碍的主要原因， 硬皮病在延长病人预期寿命方面的巨大成功 在过去20年中获得的CF导致患者现在经历 成年人以前没有遇到过的疾病。其中 疾病是骨质疏松症。CF患者似乎处于高风险状态， 骨质疏松症，由于营养，药理学和遗传因素，但 该问题的病理生理学和程度尚不清楚。患者 硬皮病将进行前瞻性随访并评估皮肤病 通过皮肤评分、硬度计读数（厚度）、基于光的活动 技术、皮肤活检、自我评估和功能状态 仪器.将对这些数据进行分析，以确定一组核心结果 硬皮病的治疗措施并经国家一级的专家小组验证， 研究人员在这种疾病。CF患者的观察性队列将 被研究。基线和2年骨密度、营养 状态和骨转换的生化标志物。一 负责人培训新临床研究者的综合计划 调查员建议。该计划包括学员采取积极和 在所描述的研究中发挥不可或缺的作用。此外，学员将 就读于生物统计学，流行病学和临床 研究技术导致硕士学位。一个独特的研讨会和一系列 东道机构的支助服务将进一步补充 培训计划。

DESCRIPTION (provided by applicant): To provide support for research projects in the field of rheumatology that will form the basis of a structured mentoring program for young investigators pursuing careers in patient-oriented clinical research. This application has two major specific research aims: 1) Develop new outcome measures for skin assessment in scleroderma for use in clinical trials and 2) Determine the prevalence and progression of osteoporosis in patients with cystic fibrosis (CF). Both projects will have direct relevance to furthering the health of the populations under study. Clinical research in scleroderma, including therapeutic trials, is greatly hampered by a lack of reliable and precise outcome measurements of disease activity. Skin thickening and fibrosis are major causes of morbidity and dysfunction for patients with scleroderma. The great success in extending the life expectancy of patients with CF gained in the last 20 years has resulted in patients now experiencing diseases as adults not formerly encountered in this population. Among these diseases is osteoporosis. Patients with CF appear to be at high risk for osteoporosis due to nutritional, pharmacologic, and genetic factors but the pathophysiology and extent of the problem is not known. Patients with scleroderma will be followed prospectively and evaluated for skin disease activity by skin scoring, durometer readings (thickness), light-based technologies, skin biopsies, self-assessments, and functional status instruments. These data will be analyzed to determine a core set of outcome measures for scleroderma. and validated by an expert panel of national researchers in this disease. An observational cohort of patients with CF will be studied. Baseline and 2-year measurements of bone density, nutritional status, and biochemical markers of bone turnover will performed. A comprehensive program for training new clinical investigators by the principal investigator is proposed. This program includes trainees taking an active and integral role in the research studies described. Additionally, trainees will be enrolled in formal coursework in biostatistics, epidemiology, and clinical research techniques leading to a master degree. A unique seminar and a series of support services at the host institution will further complement the training program.