Research Project
7679714
DESCRIPTION (provided by applicant): The purpose of this application is to measurably prevent and reduce the complications of inherited bleeding disorders among residents of Region DC - California, Hawaii, Nevada and Guam. This will be accomplished by continued implementation of diagnostic, clinical management, education, outreach, standardized surveillance, and prevention services that reduce hemophilia morbidity and mortality by 40%. Services will be conducted by the Region DCs 14 multidisciplinary Hemophilia Treatment Centers (HTC), and its regional administrative office. These entities have been part of the nearly 30-year-old U.S. bleeding disorder infrastructure that is organized into 12 regions and provide services per federal CDC, HRSA and Healthy People 2010 priorities for Americans with disabilities to live longer healthier lives. Our HTCs and regional office will: (1) Expand clinical assessment, prevention, surveillance, outreach, education, consultation, and management services for people of all ages and racial/ethnic backgrounds who have hemophilia, von Willebrand Disease and other inherited bleeding and clotting disorders in our catchment area that are directed at attaining and measuring specific outcomes to reduce complications;(2) Expand the cohort through continued recruitment and standard data collection;(3) Transition the national database from paper to electronic, from email to web-based;(4) Expand the database to collect new data related to women's ssues in coagulation, rare bleeding disorders, prophylaxis, factor utilization, thrombophilia, inhibitors, and other important clinical outcomes;(5) Design and pilot additional studies aimed at further characterizing these populations through the expanded access to a) gene-based diagnosis and b) more precise definition of the biochemical phenotype;(6) Design prevention messages and strategies, clinical trials and data sharing with other relevant agencies;and (7) Maintain a regional office which will support the HTC network and assess the efficacy of prevention services.
