Preventing Bleeding Disorder Complications thru Hemophilia Tx Ctrs in Region IX

描述(申请人提供)：本申请的目的是在DC-加利福尼亚、夏威夷、内华达和关岛地区的居民中有效地预防和减少遗传性出血性疾病的并发症。这将通过继续实施诊断、临床管理、教育、外展、标准化监测和预防服务来实现，从而将血友病发病率和死亡率降低40%。服务将由该地区的14个多学科血友病治疗中心(HTC)及其地区行政办公室提供。这些实体是已有近30年历史的美国出血障碍基础设施的一部分，该基础设施分为12个地区，根据联邦疾控中心、HRSA和健康人2010年的优先事项为美国残疾人提供服务，以延长他们的健康寿命。我们的血友病控制中心和区域办事处将：(1)为我们集水区内所有年龄和种族/民族背景的血友病、von Willebrand病和其他遗传性出血和凝血障碍患者扩大临床评估、预防、监测、外展、教育、咨询和管理服务，旨在获得和衡量特定结果，以减少并发症；(2)通过继续招募和标准数据收集，扩大队列；(3)将国家数据库从纸质到电子，从电子邮件到网络；(4)扩大数据库，以收集有关妇女在凝血、罕见出血疾病、预防、因子利用、血栓形成、抑制剂和其他重要临床结果方面的新数据；(5)设计和试点其他研究，旨在通过扩大获得a)基于基因的诊断和b)更精确的生化表型定义来进一步确定这些人群的特征；(6)设计预防信息和策略、临床试验和与其他相关机构共享数据；以及(7)维持一个地区办事处，支持HTC网络并评估预防服务的有效性。

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.