Studies in Scleroderma and Cystic Fibrosis Osteoporosis

描述(申请人提供)：为研究项目提供支持 在风湿学领域，这将构成结构化指导的基础 面向以患者为中心的临床领域的年轻研究人员的计划 研究。该应用有两个主要的具体研究目的：1)开发新的 用于临床试验的硬皮病皮肤评估的结果指标 2)确定患者骨质疏松症的患病率和进展 伴有囊性纤维化(CF)。这两个项目都将直接与 促进被研究人群的健康。中国的临床研究 硬皮病，包括治疗试验，由于缺乏治疗药物而大大受阻。 对疾病活动性的可靠和精确的结果衡量。皮肤加厚 和纤维化是患者发病和功能障碍的主要原因 硬皮病。延长患者预期寿命的巨大成功 在过去的20年中获得了CF，导致患者现在经历 作为成年人的疾病，以前在这个人群中没有遇到过。其中包括 疾病就是骨质疏松症。慢性萎缩性胃炎患者似乎有很高的风险 由营养、药物和遗传因素引起的骨质疏松症 病理生理学和问题的程度尚不清楚。患有疾病的患者 硬皮病将被前瞻性地跟踪和评估皮肤病。 按皮肤评分、硬度计读数(厚度)、基于光线的活动 技术、皮肤活检、自我评估和功能状态 乐器。将对这些数据进行分析，以确定一组核心结果 硬皮病防治措施。并由国家专家小组进行验证 这种疾病的研究人员。一组CF患者的观察性队列将 被研究。基线和两年的骨密度、营养测量 状态，并进行骨转换的生化标记物。一个 校长培训新的临床研究人员的综合计划 提出了调查员的建议。这一计划包括学员参加积极和 在所述研究研究中发挥不可或缺的作用。此外，受训人员将被 注册了生物统计学、流行病学和临床的正式课程 获得硕士学位的研究技术。一个独特的研讨会和一系列 在主办机构提供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.