Ghana-SPARCO: Ghana Sickle Pan-African Research Consortium

项目摘要 镰状细胞病（SCD）是一种以血红蛋白紊乱为特征的遗传性疾病 结构，异常内皮相互作用，全身炎症，氧化应激，以及 凝血系统这些异常是无数并发症的基础，包括急性血管闭塞性 综合征和多系统进行性慢性终末器官损伤。撒哈拉以南非洲（SSA） 不成比例的高负担，全球2500万SCD患者中估计有1500万; 75% 在所有患有SCD的婴儿中，5岁以下儿童的死亡率在50%-90%之间。这些包括 缺乏国家控制方案，缺乏管理病人的基本设施，缺乏系统的 SCD筛查，普遍缺乏循证预防干预措施的实施， 对疾病起源的误解，以及缺乏政府支持。从数量上看 尽管非洲的患者中有大量的临床信息， 用于分析的业务研究，为基于证据的医疗保健决策提供信息，以及 在欧洲大陆的SCD医疗保健政策。为此，镰刀泛非研究联合会 （SPARCo）于2017年开始在三个国家的多个地点进行登记：加纳（ 目前的U 01赠款）、尼日利亚和坦桑尼亚。在SPARCO的第一阶段，我们建立了一个合作网站， 在库马西，招募了2，915名SCD患者，包括1，453名来自我们的新生儿筛查项目的患者， 登记册，并为今后的研究建立能力。在第二阶段，我们增加了一个国内 位于首都阿克拉的卫星站点，在以下地区有完善的临床、培训和研究方案： SCD，组成加纳SPARCO。我们的总体目标是建立一个6,000人的前瞻性队列 在整个生命周期中与SCD一起生活，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 在乎我们的愿景是在加纳建立卓越的SCD研究和护理中心。我们将与 协调中心：（i）建立统一的患者登记处，（ii）整合护理标准， SCD，（iii）开展队列研究，重点评估神经血管、肾功能损害的负担和预测因素， 及感染并发症;及（iv）就以下事宜进行研究 新生儿筛查，SCD诊所青春期的保留以及制定常规 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.