Outcomes and Implementation Research Unit, Department of Nephrology and Hypertension, University of Kansas Medical Center, Kansas City, KS, USA.
Hematology Division, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA.
Haemophilia. 2020 Jan;26(1):106-116. doi: 10.1111/hae.13881. Epub 2019 Nov 26.
von Willebrand disease (VWD) is an inherited bleeding disorder caused by a quantitative or qualitative dysfunction of von Willebrand factor. Clinicians, patients and other stakeholders have many questions about the diagnosis and management of the disease.
To identify topics of highest importance to stakeholders that could be addressed by guidelines to be developed by the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF) and the World Federation of Hemophilia (WFH).
A survey to determine and prioritize topics to be addressed in the collaborative development of guidelines for VWD was distributed to international stakeholders including patients, caregivers and healthcare providers (HCPs). Representatives of the four organizations coordinated the distribution strategy. The survey focused on both diagnosis and management of VWD, soliciting 7-point Likert-scale responses and open-ended comments, in English, French and Spanish. We conducted descriptive analysis with comparison of results by stakeholder type, gender and countries' income classification for the rating questions and qualitative conventional content data analysis for the open-ended responses.
A total of 601 participants responded to the survey (49% patients/caregivers and 51% healthcare providers). The highest priority topics identified were diagnostic criteria/classification, bleeding assessment tools and treatment options for women and surgical patients. In contrast, screening for anaemia and differentiating plasma-derived therapy versus recombinant therapies received lower ratings.
This survey highlighted areas of importance to a diverse representation of stakeholders in the diagnosis and management of VWD, providing a framework for future guideline development and implementation.
血管性血友病(VWD)是一种遗传性出血性疾病,由血管性血友病因子的数量或质量功能障碍引起。临床医生、患者和其他利益相关者对该疾病的诊断和管理有许多疑问。
确定对利益相关者最重要的主题,这些主题可以通过美国血液学会(ASH)、国际血栓与止血学会(ISTH)、国家血友病基金会(NHF)和世界血友病联合会(WFH)制定的指南来解决。
一项调查旨在确定并确定在 VWD 指南的协作制定中需要解决的主题的优先级,该调查分发给包括患者、护理人员和医疗保健提供者(HCP)在内的国际利益相关者。四个组织的代表协调了分发策略。该调查侧重于 VWD 的诊断和管理,征求了 7 分李克特量表的答复和英文、法语和西班牙语的开放式回答意见。我们对评分问题进行了描述性分析,并比较了利益相关者类型、性别和国家收入分类的结果,对开放式回答进行了定性常规内容数据分析。
共有 601 名参与者对调查做出了回应(49%的患者/护理人员和 51%的医疗保健提供者)。确定的优先级最高的主题是诊断标准/分类、出血评估工具以及女性和手术患者的治疗选择。相比之下,贫血筛查和区分血浆衍生疗法与重组疗法的评级较低。
这项调查强调了在 VWD 的诊断和管理方面,对不同利益相关者的重要领域,为未来的指南制定和实施提供了框架。