Prevention of the Complications of Bleeding Disorders Through HTCS

描述（由申请人提供）：血友病是一种由凝血因子VIII或因子IX缺乏引起的遗传性出血性疾病，血管性血友病（VWD）是一种由血管性血友病蛋白减少或异常引起的遗传性出血性疾病。这些出血性疾病可导致自发性和外伤性出血。该项目的目的是通过区域血友病治疗中心（HTCs）网络预防出血性疾病的并发症。由来自DE， MD， PA， VA， WV和DC的15个HTCs组成，III区为3061名出血性疾病患者提供服务。该项目的主要研究者和重要贡献者在护理出血性疾病患者方面拥有丰富的经验，并得到多学科团队的支持。每个HTC都是一个专科、预防、诊断和治疗项目，为患有出血性疾病的儿童、青少年和成人提供评估、教育、研究、监测、外展、咨询和管理。综合护理已被证明可降低男性血友病患者的死亡率。在这一成功的基础上，并通过CDC的通用数据收集研究使用数据，该网络准备回答与遗传性出血性疾病患者最佳护理和预防策略相关的关键问题。与全国范围内的HTC系统合作，第三区域网络将通过以下具体目标促进对健康促进和疾病、伤害和残疾预防的理解：1)通过扩大第三区域HTC参与UDC研究和利用收集到的数据来解决研究问题，包括：a)农村或城市居民是否影响UDC参与和HTC资源的利用？b)中度血友病患者的关节功能是否比重度血友病患者在一级预防或改良一级预防下更差？2)维持卫生保健中心的区域网络，以便通过以下方式收集数据并实施预防战略：提供全面护理以减少出血性疾病的并发症；制定项目扩大HTC患者群体，包括女性和少数族裔；为HTC新员工和老员工提供培训资源；并为其他医疗服务提供者提供教育资源。3)通过宾夕法尼亚大学核心中心与国家核心中心网络合作，实施DC支持的区域活动，通过促进HTCs之间的沟通来实现这些项目活动和研究的目标；维持区域执行委员会；组织年度区域会议；对分包的htc进行监督；作为DC、妇幼卫生局、国家血友病基金会和卫生保健中心之间的联络人；通过全国范围内的HTC网络共享研究数据；参与疾病预防控制中心协调委员会。

DESCRIPTION (provided by applicant): Hemophilia is an inherited bleeding disorder resulting from a deficiency of coagulation factor VIII or factor IX and von Willebrand Disease (VWD) is an inherited bleeding disorder resulting from decreased or abnormal von Willebrand protein. These bleeding disorders result in both spontaneous and trauma-induced bleeding. The purpose of this project is to prevent complications of bleeding disorders through the network of Region hemophilia treatment centers (HTCs). Consisting of 15 HTCs in DE, MD, PA, VA, WV, and DC Region III serves 3,061 individuals with bleeding disorders. The principal investigator and significant contributors for this project have extensive experience in caring for individuals with bleeding disorders and are supported by a multi-disciplinary team. Each HTC is a specialty, prevention, diagnostic and treatment program providing assessment, education, research, surveillance, outreach, consultation, and management to children, adolescents, and adults with bleeding disorders. Comprehensive care has been shown to decrease mortality n men with hemophilia. Building on this success and using data through the CDC's Universal Data Collection study, this network is poised to answer key questions related to optimal care and prevention strategies for patients with inherited bleeding disorders. Working collaboratively with the nationwide system of HTCs, the Region III network will foster the understanding of health promotion and prevention of disease, injury and disability by the following Specific Aims: 1) To enhance prevention activities and improve outcomes by: expanding Region III HTC participation in the UDC study and utilizing data collected to address research questions, including: a) Does rural or urban residence impact UDC participation and utilization of HTC resources? b) Do patients with moderate hemophilia have worse joint function than patients with severe disease on primary or modified primary prophylaxis? 2) To maintain the regional network of HTCs to allow data collection and implementation of prevention strategies by: providing comprehensive care to reduce complications from bleeding disorders; developing programs to expand the HTC patient populations, including women and minorities; facilitating training resources for new and established HTC staff; and providing educational resources for other medical providers. 3) To implement DC supported regional activities through the University of Pennsylvania Core Center to collaborate with a national network of Core Centers to implement the objectives of these project activities and studies by: facilitating communication between the HTCs; maintaining a Regional Executive Committee; organizing an annual Regional Meeting; providing oversight to the subcontracting HTCs; serving as a liaison between the DC, Maternal and Child Health Bureau, National Hemophilia Foundation and HTCs; sharing research data throughout the nationwide HTC network; and participating in the CDC Coordinating Committee.