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: 7233464
• 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/7233464
• 关键词: blood coagulation disorders; clinical research; cooperative study; disease /disorder prevention /control; epidemiology; hemophilia As; human subject; medical complication; outcomes research; von Willebrand' s disease

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）：本申请的目的是衡量和减少DC地区 - 加利福尼亚州，夏威夷，内华达州和关岛的遗传出血疾病的并发症。这将通过继续实施诊断，临床管理，教育，外展，标准化监视和预防服务来实现，从而将血友病的发病率和死亡率降低40％。服务将由DCS 14个多学科血友病治疗中心（HTC）及其区域行政办公室进行。这些实体已成为近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，并根据联邦疾病预防疾护，HRSA和Healthy People提供服务，2010年美国残疾人生活更长的健康生活。我们的HTC和区域办公室将：（1）扩大临床评估，预防，监视，外展，教育，教育，咨询和管理服务，为各个年龄段的人以及种族/种族背景的人，患有血友病，冯·威勒布兰氏病以及其他遗传性疾病的血液友善和其他遗传性疾病的疾病，这些疾病在我们的背心中降低了对比的混乱局面，以降低了对比的混乱局面，以降低对比的造成对比。 （2）通过继续招聘和标准数据收集扩展队列； （3）将国家数据库从纸转换为电子，从电子邮件到基于网络； （4）扩展数据库，以收集与女性在凝血，罕见出血疾病，预防性，因子利用率，血栓形成症，抑制剂和其他重要临床结果中有关的新数据； （5）设计和试点的其他研究旨在通过扩大的访问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，与其他相关机构共享的临床试验和数据共享； （7）维护一个区域办公室，该办公室将支持HTC网络并评估预防服务的功效。