Rare disorders that are associated with an increased thrombotic risk include the antiphospholipid antibody syndromes (APS), heparin-induced thrombocytopenia (HIT), combined thrombophilic states, paroxysmal nocturnal hemoglobinuria, thrombotic thrombocytopenic purpura, and the catastrophic 'thrombotic storm'. These disorders frequently exhibit more """"""""aggressive"""""""" clinical phenotypes, affecting arterial, venous, and/or microvascular beds. Diagnostic and/or therapeutic limitations exist for each of these disorders, and prospective studies are needed to more clearly define the syndromes and develop better therapies. This application is for a multi-institutional academic center that focuses on rare thrombotic disorders, which will be developed through the following Aims: (1) Establish a Rare Disease Clinical Research Center focused on rare thrombotic disorders. Investigators from four academic centers will bring together existing registries (e.g., Antiphospholipid Syndrome Collaborative Registry) and programs (e.g., CDC-sponsored Thrombophilia Programs; Duke Center for Human Genetics) to identify and enroll patients into hypothesis-driven prospective clinical trials that focus on: . Genetic analysis of familial APS, familial APS/autoimmunity syndromes, and patients with catastrophic """"""""thrombotic storm"""""""". . Identify risk factors for thrombosis in patients with antiphospholipid antibodies and HIT. . Define the natural history of patients with elevated hepadn-platelet factor 4 antibodies after bypass. Emerging opportunities from ongoing studies will be identified that promote new research directions, projects, and translational activities that foster links between the Center and industry. (2) Develop a training program for new investigators who are interested in rare thrombotic disorders. A program will be instituted that combines opportunities in clinical management as well as epidemiologic, genetic, diagnostic, and therapeutic investigations involving patients with rare thrombotic disorders. (3) Develop a web site that promotes education and research activities involving patients with rare thrombotic disorders. The web-site will be developed with the Data Technology and Coordinating Center and other Rare Disease Research Centers and will be for patients, healthcare providers, and the general public.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
1U54HL077878-01
Application #
6745387
Study Section
Special Emphasis Panel (ZRG1-EDC-1 (50))
Program Officer
Ganguly, Pankaj
Project Start
2004-03-01
Project End
2008-12-31
Budget Start
2004-03-01
Budget End
2004-12-31
Support Year
1
Fiscal Year
2004
Total Cost
$977,718
Indirect Cost
Name
Duke University
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
044387793
City
Durham
State
NC
Country
United States
Zip Code
27705
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