Ghana-SPARCO: Ghana Sickle Pan-African Research Consortium

项目总结 镰状细胞病(SCD)是一种以血红蛋白紊乱为特征的隐性遗传性疾病 结构，异常的内皮相互作用，全身炎症，氧化应激，和激活 凝血系统。这些异常导致了无数的并发症，包括急性血管闭塞。 综合征和多系统进行性慢性终末器官损害。撒哈拉以南非洲(SSA)承担着 全球2500万SCD患者中估计有1500万人背负着不成比例的负担；75% 在所有出生患有SCD的婴儿中，5岁以下儿童的死亡率在50%-90%之间。这些措施包括 缺乏国家控制计划，缺乏管理病人的基本设施，缺乏系统性 SCD筛查，普遍缺乏基于证据的预防性干预措施，文化 对疾病起源的误解，以及缺乏政府支持。仅仅从数量上看 在非洲的患者中，存在着获取大量临床信息的潜力 用于分析的运筹性研究，为基于证据的医疗决策提供信息，以及 非洲大陆慢性阻塞性肺病的医疗政策。为此，镰刀泛非研究联盟 (Sparco)于2017年开始在三个国家的多个地点进行登记：加纳(申请 目前的U01赠款)、尼日利亚和坦桑尼亚。在Sparco的第一阶段，我们建立了一个协作站点 在库马西招募了2,915名SCD患者，其中包括1,453名来自我们的新生儿筛查计划进入 登记，并为今后的研究建立能力。在第二阶段中，我们添加了一个国家/地区内 位于首都阿克拉的卫星站点，该站点在 SCD，组建加纳-斯巴科。我们的首要目标是建立一个由6000人组成的预期队列 一生中患有SCD的人有双重目的：1)多变的决定因素 在资源有限的情况下SCD的急性和慢性并发症的表现，以及2)进行 实施研究以解决、超越和整合最佳的基于资源的临床标准 关心。我们的愿景是在加纳建立SCD研究和护理的卓越中心。我们将与 协调中心；(1)建立统一的病人登记制度，以(2)统一护理标准 SCD，(3)进行队列研究，重点评估神经血管、肾脏的负担和预测因素 和传染性疾病的终身感染并发症&(Iv)开展以下方面的实施研究 新生儿筛查，在SCD诊所保留青春期&制定常规实施战略 整合SCD的预防性干预措施。达到这些基准将有助于建立 在SSA研究网络中开发SCD的基础设施。

PROJECT SUMMARY Sickle Cell Disease (SCD) is a recessively inherited disorder characterized by disordered hemoglobin structure, aberrant endothelial interactions, systemic inflammation, oxidant stress, and activation of the coagulation system. These abnormalities underlie a myriad of complications including acute vaso-occlusive syndromes and a multi-system progressive chronic end-organ damage. Sub-Saharan Africa (SSA) bears a disproportionately higher burden with an estimated 15 million of the world’s 25 million SCD individuals; 75% of all babies born with SCD; & mortality rate for children under 5 range between 50%-90%. These include absence of National control programs, dearth of basic facilities to manage patients, absence of systematic screening for SCD, pervasive lack of implementation of evidence-based preventative interventions, cultural misconceptions about the genesis of the disease, and lack of governmental support. By the sheer numbers of patients in Africa, there is a potential for large volumes of clinical information to be harvested though operational research for analyses to inform evidence-based decision-making on healthcare, as well as healthcare policies for SCD on the continent. To this end, the Sickle Pan-African Research Consortium (SPARCo) in 2017 started a registry across multiple sites in three countries: Ghana (an applicant for the current U01 grant), Nigeria, and Tanzania. In the first phase of SPARCO, we established a collaborative site in Kumasi and enrolled 2,915 SCD patients including 1,453 from our Newborn screening program into a registry, and built capacity for future research studies. In this second phase, we have added an in-country Satellite site in the capital city Accra, which has well developed clinical, training and research programs in SCD, to form Ghana-SPARCO. Our overarching goal is to establish a prospective cohort of 6,000 individuals living with SCD across the lifespan with a dual purpose of understanding: 1) the determinants of the protean manifestations of acute and chronic complications of SCD in a resource-limited setting, and 2) to conduct implementation research to address, surmount & integrate the best resource-based standards of clinical care. Our vision is to establish hubs of excellence for research and care for SCD in Ghana. We will work with the Coordinating Center to; (i) establish a harmonized patient registry to, (ii) integrate standards of care for SCD, (iii) conduct cohort studies focusing on an assessment of burden and predictors of neurovascular, renal and infectious complications of SCD across the lifespan & (iv) conduct implementation research studies on newborn screening, retention of adolescence in SCD clinics & develop an implementation strategy for routine integration of preventative interventions for SCD. Achieving these benchmarks will help establish the infrastructure to develop a SCD in SSA Research Network.