Developing a Sickle Africa Data Coordinating Center (SADaCC)

项目摘要/摘要(30行) 据估计，每年有30.58万名婴儿出生时患有镰状细胞病(SCD) 全球近75%的新生儿发生在撒哈拉以南非洲(SSA)。尽管如此 发病率高，但目前在任何SSA国家都没有有效的公共卫生方案 专注于SCD。因此，SSA中高达90%的SCD婴儿被认为是 五年后死亡。来自西方的证据表明，干预的制度 由于新生儿筛查和青霉素预防被认为是减少了~70%的 儿童SCD病死率。然而，中国成年SCD患者的死亡率 由于其他令人衰弱的并发症，美国的病情没有改善。因此，有一个重大的 需要研究以帮助开发全身SCD的有效治疗方法 这个世界。纵向队列研究是最科学、最严格的方法 对风险因素、健康和疾病后果的了解。年疾病患病率较高 SSA有望促进流行病学、转化性和临床研究 对SCD的研究，类似于对心脏的Framingham研究。 该U24应用程序正在寻求建立一个名为DCC的数据协调中心 镰刀非洲数据协调中心，以支持一个 非洲的SCD伙伴网络。这一总体目标将分四个阶段实现 具体目标：目标1：提供全面协调和行政支助， 将包括制定和维护操作程序；创建和 SCD本体的管理。目标2：创建并确保维护 集中化的电子镰刀状血红蛋白病数据库将包括开发 议定书，包括适当的同意程序和提供数据 管理和生物信息学技能。目标3：提供生物统计学、研究 开发、分析和医疗方面的专业知识。目标4：提供2年的开发 第一阶段，一年试点/规划阶段和一年实施阶段。 我们相信，作为协调中心，我们的团队有a)以前的经验， H3非洲联盟，b)非洲大学杰出的体制环境 开普敦(UCT)，c)UCT临床研究中心在 生物信息学能力发展方面的研究/数据管理和H3ABioNet，以及d) 我们在基础、转化和临床研究和培训方面的记录侧重于SCD SSA，是为SCD联盟建立最有效的DCC的好兆头。

Project Summary/Abstract (30 lines) It is estimated that 305,800 babies are born each year with Sickle Cell Disease (SCD) worldwide with nearly 75% of the births occurring in sub-Saharan Africa (SSA). Despite this high incidence, there are currently no effective public health programs in any SSA country focused on SCD. As a consequence, up to 90% of infants with SCD in SSA are believed to die by five years. Evidences from the West indicate that the institution of interventions such as newborn screening and penicillin prophylaxis are credited with the ~70% reduction in mortality rate among children with SCD. However, the death rate in adult SCD patients in the US has not improved, due to additional debilitating complications. There is therefore a major need for research to help develop effective therapies across the life span for SCD in all parts of the world. Longitudinal cohort studies are the most scientifically rigorous methods in understanding of, risk factors, and health and disease outcomes. High disease prevalence in SSA would be expected to facilitate epidemiologic, translational, and clinical research studies on SCD, similar to Framingham Study for the heart. This U24 application is seeking to establish a Data Coordinating Center (DCC) to be called the Sickle Africa Data Coordinating Center (SADaCC), to support the activities of a companion SCD Network in Africa. This overall objective will be accomplished by four specific aims: Aim 1: To provide overall coordination and administrative support that will include development and maintenance of operating procedures; creation and management of SCD Ontology. Aim 2: To create and ensure maintenance of a centralized, electronic sickle hemoglobinopathy database that will include development of protocols, including appropriate consenting procedures and provision of data management and bioinformatics skills. Aim 3: to provide biostatistical, study development, analytic and medical expertise. Aim 4: to provide 2 year development phase, and 1 year pilot/planning phase and a 1 year implementation phase. We believe that our team’s a) prior experience, as a coordinating center, of the activities of the H3Africa Consortium, b) the outstanding institutional environment at the University of Cape Town (UCT), c) the long experience of the UCT Clinical Research Centre in research/data management and H3ABioNet in bioinformatics capacity development, and d) our track record in basic, translational and clinical research, and training focused on SCD in SSA, augurs well to establish the most effective DCC for the SCD consortium.