Sickle Pan African Research Consortium NigEria (SPARC-NEt)

尼日利亚是世界上镰状细胞病（SCD）发病率最高的国家，估计有15万婴儿 每年出生。非洲患有SCD的儿童特别容易受到感染，约50%-90%的儿童死亡 在五岁之前。通过早期诊断和支持性治疗， 青霉素预防和羟基脲（HU）治疗，特别是对那些有中风风险的人。但这些 干预措施尚未在尼日利亚的SCD患者中广泛实施。镰刀泛非研究 在加纳、尼日利亚和坦桑尼亚设有合作点的SPARCO联盟由NIH/ NHLBI资助 在2017年建立SCD未来研究的基础设施。SPARCO在尼日利亚的工厂位于 阿布贾大学镰状细胞病研究与培训卓越中心（CESRTA）。上 在授予阶段，我们建立了与临床和数据协调相关的管理结构， 中心，并积极参加联合工作组的活动。我们获得并保持了道德 在“镰刀非洲”数据协调中心的指导下建立了一个REDCap数据库 （SADaCC）;并使用统一的病例报告表在19个研究中心成功招募了4，687名患者， 与尼日利亚镰状细胞支持协会合作。（图）我们提供了专门的培训， 对915名卫生保健工作者（包括初级卫生保健工作者）进行SCD研究和卫生保健管理 初级保健中心。我们开发了临床研究硕士课程和研究生课程， 生物信息学文凭，以解决已确定的差距。我们分析了登记处的初步数据， 描述了SPARCO尼日利亚队列中SCD的临床表型，并进行了实施研究 在新生儿。在这些成功的基础上，我们现在的目标是维持、加强和发展SCD网络 在非洲，以推进与SCD相关的流行病学、转化和临床研究和政策， 镰刀泛非研究联盟尼日利亚（SPARC-NEt）队列研究。我们的战略是增加 * 通过以下方式将SPARC-Net SCD队列人数增加到9 000人：a）再招募2 000人， 招募地点和B）扩大新生儿筛查以建立1000名SCD患者的出生队列。我们将 扩展、实施和评估SPARCO护理标准（SoC）指南的使用，以进行随访 SPARC-NEt研究中心SCD患者的管理，并确定培训的影响， 与卫生工作者、父母和支持团体就个人的总体健康结果进行接触， 尼日利亚的SCD。此外，我们描述了三个可能的实施和队列研究， 包括a）使用以下方法评估患者特征和临床事件的发生率 SPARCO SoC，B）评价HU使用对患者临床和血液学结局的影响， SPARC-Net队列，以及c）使用基因组方法研究 SPARC-Net SCD出生队列中的肠道微生物组、HbF水平和血管闭塞危象频率。

Nigeria has the highest prevalence of sickle cell disease (SCD) in the world with an estimated 150,000 babies born annually. Children with SCD in Africa are particularly susceptible to infections, with about 50%-90% dying before five years of age. This burden can be substantially reduced by early diagnosis and supportive care with penicillin prophylaxis, and hydroxyurea (HU) treatment, especially for those at risk of stroke. However, these interventions are yet to be widely implemented in patients with SCD in Nigeria. The Sickle Pan African Research Consortium (SPARCO), with collaborative sites in Ghana, Nigeria and Tanzania was funded by the NIH/ NHLBI in 2017 to establish the infrastructure for future research in SCD. The SPARCO Site in Nigeria is set up at the Centre of Excellence for Sickle Cell Disease Research &Training, University of Abuja (CESRTA). In the first phase of the award, we established the management structure in relation to the Clinical and Data Coordinating Centres and participated actively in consortium working groups’ activities. We obtained and maintained ethical approvals; established a REDCap database under the guidance of the Sickle Africa Data Coordinating Centre (SADaCC); and successfully recruited 4,687 patients across 19 sites using uniform case report forms in collaboration with the Sickle Cell Support Society of Nigeria. (Figure) We delivered specialized training in research and health care management of SCD to 915 health care workers including those in primary health care centres (PHCs). We developed a curriculum for a Master’s degree in Clinical Research and a Postgraduate Diploma in Bioinformatics to address identified gaps. We analyzed preliminary data from the registry and described the clinical phenotypes of SCD in SPARCO Nigeria cohort and conducted an implementation study on newborn. Building on these successes, we are now aiming to sustain, enhance and grow the SCD Network in Africa in order to advance SCD-related epidemiological, translational and clinical research and policy through the Sickle Pan African Research Consortium NigEria (SPARC-NEt) Cohort Study. Our strategy is to increase the SPARC-Net SCD Cohort to 9,000 by: a) recruiting additional 2,000 individuals and bringing in six additional recruitment sites and b) expanding newborn screening to establish a birth cohort of 1000 SCD patients. We will expand, implement, and evaluate the use of the SPARCO Standards of Care (SoC) guidelines for the follow up and management of patients with SCD in the SPARC-NEt Sites and determine the impact of training of, and engagement with, health workers, parents and support groups on the overall health outcomes for individuals with SCD in Nigeria. Additionally, we described three potential implementation and cohort studies that could be adopted by the consortium including a) assess patient characteristics and incidence of clinical events using SPARCO SoC, b) evaluate the impact of HU utilization on clinical and haematological outcomes in patients within the SPARC-Net cohort, and c) using genomic approaches to investigate the association between changes in gut microbiome, HbF levels and frequency of vaso-occlusive crisis in SPARC-Net SCD birth cohort.