Preventing Bleeding Disorder Complications thru Hemophilia Tx Ctrs in Region IX

通过 IX 区的血友病 Tx Ctr 预防出血性疾病并发症

• 批准号: 7279183
• 负责人: Diane J Nugent
• 金额: $ 85万
• 依托单位: CHILDREN'S HOSPITAL OF ORANGE COUNTY
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-09-30 至 2011-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7279183
• 关键词: Preventing; Bleeding; Disorder; Complications; thru

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.

描述（由申请人提供）：本申请的目的是显着预防和减少华盛顿特区（加利福尼亚州、夏威夷州、内华达州和关岛）居民的遗传性出血性疾病的并发症。这将通过持续实施诊断、临床管理、教育、推广、标准化监测和预防服务来实现，将血友病发病率和死亡率降低 40%。服务将由地区 DC 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。这些实体是拥有近 30 年历史的美国出血性疾病基础设施的一部分，该基础设施分为 12 个地区，并根据联邦 CDC、HRSA 和 Healthy People 2010 优先事项提供服务，帮助美国残疾人过上更长久、更健康的生活。我们的 HTC 和区域办事处将： (1) 为我们学区内所有年龄和种族/族裔背景的血友病、冯维勒布兰德病和其他遗传性出血和凝血疾病患者扩大临床评估、预防、监测、外展、教育、咨询和管理服务，旨在实现和衡量具体结果以减少并发症； (2) 通过持续招募和标准数据收集扩大队列； (3) 将国家数据库从纸质转变为电子、从电子邮件转变为网络； (4) 扩大数据库，收集与妇女凝血、罕见出血性疾病、预防、因子利用、血栓形成倾向、抑制剂和其他重要临床结局相关的新数据； (5) 设计和试点更多研究，旨在通过扩大 a) 基于基因的诊断和 b) 更精确的生化表型定义来进一步表征这些人群； （6）设计预防信息和策略、临床试验以及与其他相关机构共享数据； (7) 维持一个区域办事处，该办事处将支持 HTC 网络并评估预防服务的有效性。