Developing Primary Care Algorithms for Early Identification of Young Women with von Willebrand Disease

开发初级保健算法以早期识别患有冯维勒布兰德病的年轻女性

基本信息

批准号：
10673876
负责人：
SARAH H. OBRIEN
金额：
$ 62.85万
依托单位：
RESEARCH INST NATIONWIDE CHILDREN'S HOSP
依托单位国家：
美国
项目类别：
财政年份：
2022
资助国家：
美国
起止时间：
2022-08-01 至 2025-07-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10673876
关键词：
20 year old Adolescent Adult Affect Algorithms Assessment tool Blood Coagulation Disorders Caregivers Caring Child Childbirth Clinic Clinical Clinical Research Clinical Trials Data Diagnosis Early Diagnosis Early identification Enrollment Event Female Ferritin Foundations Frequencies Future General Population Goals Guidelines Health Health Personnel Hematology Hemophilia A Hemorrhage Hemostatic function Hysterectomy Incidence International Intervention Interview Iron Iron deficiency anemia Laboratories Measurement Measures Menorrhagia Menstruation Mission Morbidity - disease rate Operative Surgical Procedures Outcome Parents Participant Patients Postpartum Period Primary Care Quality of life Reach, Effectiveness, Adoption, Implementation, and Maintenance Recording of previous events Reference Values Research Research Personnel Screening procedure Severities Societies Specificity Structure Symptoms Technology Testing Thrombosis Time United States Validation Visit Woman Work experience girls health related quality of life implementation framework innovation iron deficiency medical specialties meetings mortality patient population point of care primary care clinic primary care setting prospective recruit research study screening screening guidelines standard of care study population success treatment center von Willebrand Disease young woman

项目摘要

PROJECT SUMMARY / ABSTRACT Bleeding disorders in women remain under-recognized and under-treated. Although expert guidelines recommend screening adolescents with heavy menstrual bleeding (HMB) for von Willebrand Disease (VWD), our team has identified that under-screening remains widespread. Recent advances in VWD screening include the validation of bleeding assessment tools (BATs), with the International Society of Thrombosis and Haemostasis BAT (ISTH-BAT) serving as the current standard. However, the reference range of the ISTH-BAT score for young women is unknown, and there is a lack of research examining the accuracy of BATs in primary care settings. Our team has also shown that iron deficiency is common in young women with HMB, and while point-of-care (POC) ferritin technology is newly available, its utility as a screening tool for VWD in women with HMB has not been evaluated. The primary objective of this R01 application is to compare the validity of three screening strategies to identify young women with HMB who have an underlying diagnosis of VWD: 1) BAT, 2) POC ferritin, and 3) combined BAT + POC ferritin. Our rationale for this research is that early diagnosis of VWD in women with HMB can alleviate morbidity and mortality by enabling health care providers to optimally manage HMB and develop care plans for future surgical events and childbirth, thus decreasing the incidence and severity of bleeding events. Building upon our expertise in BATs, VWD, and past success in the recruitment of young women for HMB studies, we will achieve our objectives through the following specific aims: 1) establish the reference range of the ISTH-BAT in a general population of post-menarchal females 10-20 years of age, 2) compare the validity of the following screening tools (BAT, ferritin, BAT+ferritin) in identifying young women with HMB and underlying VWD, and 3) assess the feasibility of a primary care VWD screening algorithm, as well as patient, caregiver, and clinician experience. Our study population will include ~1,800 post-menarchal females presenting for care at five primary care clinics. Participants will complete the BAT and POC ferritin during their clinical visit. Those meeting the study definition of HMB will undergo laboratory testing for VWD. We will collect feasibility measurements (i.e., participation rate, time to complete screening algorithm). We will perform semi- structured interviews with a subset of participants, parents, and health care providers to explore domains such as the acceptability, appropriateness, and impact of VWD screening on clinic workflows. Our central hypothesis is that BAT + ferritin will have higher specificity for VWD than BAT or POC ferritin alone. Our project is innovative because it shifts the study of young women with bleeding disorders from the status quo of specialized hematology centers to the primary care settings in which young women with HMB initially present. The studies proposed here will serve as a foundation for comparing a VWD primary care screening algorithm against standard of care in a prospective clinical trial to measure its effect on health outcomes such as initiation of HMB and iron deficiency treatment, specialty referral, laboratory screening for underlying bleeding disorders, and patient quality of life.

项目摘要 /摘要妇女的出血障碍仍然不足以识别和治疗。虽然专家指南建议对von Willebrand疾病（VWD）的重度大量出血（HMB）的青少年筛查，我们的团队已经确定屏幕不足仍然很普遍。 VWD筛查的最新进展包括验证出血评估工具（蝙蝠），国际血栓形成学会和血液蝙蝠（ISTH-BAT）作为当前标准。但是，ISTH蝙蝠的参考范围年轻女性的分数未知，并且缺乏研究蝙蝠在初级蝙蝠的准确性护理设置。我们的团队还表明，在HMB的年轻女性中，铁缺乏症很常见，而新近可用的服务点（POC）铁蛋白技术，它是VWD的筛查工具 HMB尚未评估。此R01应用的主要目的是比较三个的有效性筛选识别具有VWD基础诊断的HMB年轻女性的策略：1）BAT，2） POC铁蛋白和3）BAT + POC铁蛋白组合。我们对这项研究的理由是早期诊断为VWD 在患有HMB的女性中，可以通过使医疗保健提供者最佳管理来减轻发病率和死亡率 HMB并制定了未来手术事件和分娩的护理计划，从而降低了发病率和严重性出血事件。以我们在蝙蝠，VWD的专业知识为基础，并在招募年轻的招募方面取得了成功妇女进行HMB研究，我们将通过以下特定目的实现我们的目标：1）确定在年轻女性的一般人群中，ISTH蝙蝠的参考范围10-20岁，2）比较以下筛查工具（蝙蝠，铁蛋白，蝙蝠+铁蛋白）的有效性 HMB和基础VWD，以及3）评估初级保健VWD筛选算法的可行性，以及病人，照顾者和临床医生的经验。我们的研究人群将包括约1,800名男性后女性在五个初级保健诊所提供护理。参与者将完成蝙蝠和POC铁蛋白临床访问。符合HMB的研究定义的人将对VWD进行实验室测试。我们将收集可行性测量（即参与率，完成筛选算法的时间）。我们将执行半与参与者，父母和医疗保健提供者的一部分结构化访谈，以探索此类领域作为VWD筛查对诊所工作流的可接受性，适当性和影响。我们的中心假设是BAT +铁蛋白的VWD特异性比仅BAT或POC铁蛋白具有更高的特异性。我们的项目是创新的因为它转移了患有流血疾病的年轻女性的研究以HMB最初出现的年轻妇女为中心。研究提出这将是将VWD初级保健筛查算法与护理标准进行比较的基础在一项前瞻性临床试验中，以衡量其对HMB和铁缺乏症等健康结果的影响治疗，专业推荐，潜在出血疾病的实验室筛查以及患者的生活质量。