Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.
Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.
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DOI：
10.1111/hae.14588
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发表时间：
2022-09
期刊：
影响因子：
--
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中科院分区：
文献类型：
--
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数十年的遗传性出血性疾病（BD）研究将严重血友病从儿童杀手转变为经济发达国家许多人整个生命周期的疾病。然而，健康公平，不受疾病并发症影响的生活，对于大多数遗传性BD（PWIBD）患者来说仍然是不可想象的。国家血友病基金会（NHF）和美国血栓形成和止血网络（ATHN）承担了社区驱动的美国（US）遗传性出血性疾病研究国家蓝图的开发工作，以改变所有PWIBD及其护理人员的经验。进行了广泛的社区咨询，以确定对PWIBD和那些爱和关心他们的人最重要的问题。多学科专家团队将这些关键需求领域提炼为优先研究问题，并确定了追求这些问题所需的资源和基础设施。举行了一次首脑会议，以收集反馈意见，并通报详细的蓝图。社区优先研究领域分为三大类：遗传性BD的共同问题，个体疾病的特定问题，以及基础设施和能力问题。NHF科学研究状况峰会讨论了六个工作组从社区优先事项中得出的研究问题，为起草未来几十年的研究蓝图提供了重要投入。继承BD社区走到一起，制定了美国国家遗传性出血性疾病研究蓝图，致力于改变所有PWIBD的生活，包括为最罕见的疾病和代表性不足的人群提供创新解决方案。
Decades of inherited bleeding disorders (BD) research transformed severe haemophilia from a childhood killer to a disorder managed across a full lifespan for many in economically developed countries. Health equity, a life unimpaired by disease complications, however, remains unimaginable for most people with an inherited BD (PWIBD). The National Hemophilia Foundation (NHF) and American Thrombosis and Hemostasis Network (ATHN) undertook the development of a community‐driven United States (US) National Blueprint for Inherited Bleeding Disorders Research to transform the experience of all PWIBD and those who care for them. Extensive community consultations were conducted to identify the issues most important to PWIBD and those who love and care for them. Expert multidisciplinary teams distilled these key areas of need into prioritised research questions, and identified the resources and infrastructure required to pursue them. A summit was held to gather feedback and inform the detailed blueprint. Community‐prioritised research areas fell into three broad categories: issues common across inherited BDs, those specific to individual disorders, and issues of infrastructure and capacity. NHF State of the Science Research Summit discussions of the research questions derived from the community priorities by six working groups provided important input for the drafting of the research blueprint for the coming decades. The inherited BD community came together to develop the US National Blueprint for Inherited Bleeding Disorders Research dedicated to transforming the lives of all PWIBD including innovating solutions for the rarest disorders and under‐represented populations.
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DOI：
10.1111/hae.13862
发表时间：
2020-01
期刊：
Haemophilia : the official journal of the World Federation of Hemophilia
影响因子：
--
作者：
Skinner MW;Nugent D;Wilton P;O'Mahony B;Dolan G;O'Hara J;Berntorp E
通讯作者：
Berntorp E
影响因子：
3.9
作者：
O'Mahony, B.;Dolan, G.;Goodman, C.
通讯作者：
Goodman, C.
影响因子：
3.9
作者：
Skinner, Mark W.;Dolan, Gerry;O'Mahony, Brian
通讯作者：
O'Mahony, Brian
影响因子：
4.6
作者：
Weyand AC;James PD
通讯作者：
James PD
影响因子：
10.4
作者：
Evatt, B. L.
通讯作者：
Evatt, B. L.