Rheumatic Heart Disease Research and Screening in Nepal: A Feasibility Study

项目摘要/摘要 风湿性心脏病(RHD)在中低收入人群中仍然是一个重大的公共卫生问题 全球死亡人数超过300,000人。风湿性心脏病是急性风湿热的后遗症 (ARF)由A组链球菌(GAS)引起。苄星青霉素预防(BPP)，通过三种途径。 每周肌肉注射苄星青霉素G，仍然是延迟和 通过预防反复的气体感染来预防ARF和RHD患者的后遗症。两大 对这一努力的最佳利用存在障碍。首先，尽管证实了BPP的好处，但BPP的遵守仍然 很低。不幸的是，人们对BPP坚持的相关因素知之甚少。即使是在 像尼泊尔这样的国家，有一项全国性的免费BPP计划，为大约6000名患者提供服务，但没有 一个强大的系统来前瞻性地跟踪和研究这些患者。其次，很大程度上是因为RHD最初是 没有症状，只有一小部分会受益的人参加了BPP计划。这是一个稀缺的 关于潜在高收益方法的信息和证据，如筛查儿童的一级亲属 风湿性心脏病患者识别可能受益于BPP的无症状的早期风湿性心脏病患者。我们将联合起来 华盛顿大学的资源和专业知识与我们现有合作伙伴的资源和专业知识 尼泊尔主要医院(Manmohan心胸、血管和移植中心和Dhulikhel医院) 首先创建一个易于使用、可扩展、全面的电子风湿性心脏病登记系统，并登记BPP患者 (n=1000)。在目标1中，我们将检查患者的社会人口学、临床和 与卫生服务相关的特征与遵守BPP有关。我们将使用BPP注册表 收集重要的协变量信息和坚持结果，以实现这一目标。在目标2中，我们将 确定筛查已知风湿性心脏病患者一级亲属的可行性。我们将首先邀请- 50名已知风湿性心脏病患者(已在BPP登记)的程度亲属(n=150)进行超声心动图检查 风湿性心脏病的筛查。我们会评估完成甄别工作所需的参与率、时间和费用。 每个参与者；并估计这些一级亲属风湿性心脏病的患病率。这些都是至关重要的初步结果 设计一项研究以确定筛查方法的有效性和成本效益所需的数据 重点关注一级亲属。我们的方案利用了世界上最大的风湿性心脏病患者池之一 建立健全的RHD相关质量改进和研究平台，为其奠定坚实的基础 对风湿性心脏病的风险、预防和实施进行更大规模的流行病学、干预性和实施性研究 治疗。

PROJECT SUMMARY/ABSTRACT Rheumatic Heart Disease (RHD) remains a significant public health problem in lower and middle-income countries (LMIC), accounting for over 300,000 deaths world-wide. RHD is a sequela of Acute Rheumatic Fever (ARF) caused by Group A Streptococcus (GAS). The Benzathine Penicillin Prophylaxis (BPP), through tri- weekly intramuscular injection of benzathine penicillin G, remains the mainstay of intervention to delay and prevent the sequelae among ARF and RHD patients by preventing repeat GAS infections. Two major obstacles exist to the optimal utilization of this effort. First, despite proven benefits, BPP adherence remains low. Unfortunately, there is very little knowledge on the factors associated with BPP adherence. Even in countries, like Nepal, which has a nation-wide free BPP program serving about 6000 patients, there is absence of a robust system to prospectively track and study these patients. Second, largely because RHD is initially asymptomatic, only a fraction of those who would benefit are enrolled in BPP programs. There is a dearth of information and evidence on potentially high-yield approaches like the screening of first-degree relatives of RHD patients to identify asymptomatic, early-stage RHD patients who may benefit from BPP. We will combine the resources and expertise at the University of Washington with those from our existing partners at two leading Nepali hospitals (Manmohan Cardiothoracic, Vascular and Transplant Center and Dhulikhel Hospital) to first create an easy to use, scalable, comprehensive electronic RHD registry and enroll BPP patients (n=1000) from these sites. In Aim 1, we will examine whether patient socio-demographic, clinical, and health services-related characteristics are associated with BPP adherence. We will use the BPP registry to collect important covariate information and adherence outcomes to address this aim. In Aim 2, we will determine the feasibility of screening first-degree relatives of known RHD patients. We will invite first- degree relatives (n=150) of 50 known RHD patients (enrolled in the BPP registry) for echocardiographic screening for RHD. We will assess the participation rate; time and cost required to complete the screening of each participant; and estimate the prevalence of RHD in these first degree relatives. These are vital preliminary data needed to design a study to determine the efficacy and cost-effectiveness of a screening approach that focuses on first-degree relatives. Our proposal harnesses one of the largest RHD patient pools in the world for establishing a robust RHD-related quality improvement and research platform that serves as a solid foundation for conducting larger epidemiologic, interventional, and implementation studies on RHD risk, prevention, and treatment.