Studies in Scleroderma and Cystic Fibrosis Osteoporosis

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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.