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.

Agency
National Institute of Health (NIH)
Institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Type
Midcareer Investigator Award in Patient-Oriented Research (K24)
Project #
5K24AR002224-04
Application #
6780976
Study Section
Special Emphasis Panel (ZAR1-TAS-B (M1))
Program Officer
Serrate-Sztein, Susana
Project Start
2001-07-18
Project End
2006-06-30
Budget Start
2004-07-01
Budget End
2005-06-30
Support Year
4
Fiscal Year
2004
Total Cost
$124,620
Indirect Cost
Name
Boston University
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
604483045
City
Boston
State
MA
Country
United States
Zip Code
02118
Rhee, Rennie L; Davis, John C; Ding, Linna et al. (2018) The Utility of Urinalysis in Determining the Risk of Renal Relapse in ANCA-Associated Vasculitis. Clin J Am Soc Nephrol 13:251-257
Berti, Alvise; Warner, Roscoe; Johnson, Kent et al. (2018) Brief Report: Circulating Cytokine Profiles and Antineutrophil Cytoplasmic Antibody Specificity in Patients With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis. Arthritis Rheumatol 70:1114-1121
Wallace, Zachary S; Miloslavsky, Eli M; Cascino, Matthew et al. (2017) Effect of Disease Activity, Glucocorticoid Exposure, and Rituximab on Body Composition During Induction Treatment of Antineutrophil Cytoplasmic Antibody-Associated Vasculitis. Arthritis Care Res (Hoboken) 69:1004-1010
Clain, J M; Hummel, A M; Stone, J H et al. (2017) Immunoglobulin (Ig)M antibodies to proteinase 3 in granulomatosis with polyangiitis and microscopic polyangiitis. Clin Exp Immunol 188:174-181
Baker, Joshua F; Putman, Melissa S; Herlyn, Karen et al. (2016) Body composition, lung function, and prevalent and progressive bone deficits among adults with cystic fibrosis. Joint Bone Spine 83:207-11
Fussner, Lynn A; Hummel, Amber M; Schroeder, Darrell R et al. (2016) Factors Determining the Clinical Utility of Serial Measurements of Antineutrophil Cytoplasmic Antibodies Targeting Proteinase 3. Arthritis Rheumatol 68:1700-10
Unizony, Sebastian; Villarreal, Miguel; Miloslavsky, Eli M et al. (2016) Clinical outcomes of treatment of anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis based on ANCA type. Ann Rheum Dis 75:1166-9
Putman, Melissa S; Baker, Joshua F; Uluer, Ahmet et al. (2015) Trends in bone mineral density in young adults with cystic fibrosis over a 15 year period. J Cyst Fibros 14:526-32
Miloslavsky, E M; Specks, U; Merkel, P A et al. (2015) Outcomes of nonsevere relapses in antineutrophil cytoplasmic antibody-associated vasculitis treated with glucocorticoids. Arthritis Rheumatol 67:1629-36
Geetha, Duvuru; Specks, Ulrich; Stone, John H et al. (2015) Rituximab versus cyclophosphamide for ANCA-associated vasculitis with renal involvement. J Am Soc Nephrol 26:976-85

Showing the most recent 10 out of 63 publications