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.

项目概要/摘要女性出血性疾病仍未得到充分认识和治疗。尽管专家指导建议对月经出血量较多 (HMB) 的青少年进行血管性血友病 (VWD) 筛查，我们的团队发现筛查不足的现象仍然普遍存在。 VWD 筛查的最新进展包括与国际血栓形成协会合作验证出血评估工具 (BAT) 止血最佳可行技术 (ISTH-BAT) 作为当前标准。然而，ISTH-BAT 的参考范围年轻女性的分数尚不清楚，也缺乏检验小学 BAT 准确性的研究护理设置。我们的团队还表明，患有 HMB 的年轻女性缺铁很常见，而护理点 (POC) 铁蛋白技术是最新推出的，可作为患有 VWD 的女性的筛查工具 HMB 尚未评估。此 R01 应用程序的主要目标是比较三种方法的有效性用于识别患有 HMB 且潜在 VWD 诊断的年轻女性的筛查策略：1) BAT，2) POC 铁蛋白，以及 3) 组合的 BAT + POC 铁蛋白。我们进行这项研究的理由是 VWD 的早期诊断患有 HMB 的女性可以通过使医疗保健提供者能够最佳地管理来降低发病率和死亡率 HMB 并为未来的手术事件和分娩制定护理计划，从而降低发病率和严重程度出血事件。基于我们在 BAT、VWD 方面的专业知识以及过去在招募年轻人方面的成功经验女性进行 HMB 研究，我们将通过以下具体目标来实现我们的目标： 1) 建立 ISTH-BAT 在 10-20 岁初潮后女性一般人群中的参考范围，2) 比较以下筛查工具（BAT、铁蛋白、BAT+铁蛋白）在识别年轻女性时的有效性 HMB 和基础 VWD，以及 3) 评估初级保健 VWD 筛查算法的可行性，以及患者、护理人员和临床医生的经验。我们的研究人群将包括约 1,800 名初潮后女性在五个初级保健诊所接受护理。参与者将在学习期间完成 BAT 和 POC 铁蛋白测试临床访问。符合 HMB 研究定义的患者将接受 VWD 实验室检测。我们将收集可行性测量（即参与率、完成筛选算法的时间）。我们将进行半对一部分参与者、家长和医疗保健提供者进行结构化访谈，以探索诸如 VWD 筛查的可接受性、适当性以及对临床工作流程的影响。我们的中心假设与单独使用 BAT 或 POC 铁蛋白相比，BAT + 铁蛋白对 VWD 具有更高的特异性。我们的项目很创新因为它改变了对患有出血性疾病的年轻女性的研究，脱离了专业血液学的现状中心到最初患有 HMB 的年轻女性所在的初级保健机构。提出的研究这里将作为将 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测量其对健康结果的影响，例如开始 HMB 和缺铁治疗、专科转诊、潜在出血性疾病的实验室筛查以及患者的生活质量。