Sickle Pan African Research Consortium NigEria (SPARC-NEt)

尼日利亚是世界上镰状细胞病(SCD)患病率最高的国家，估计有15万婴儿 每年出生一次。在非洲，患有SCD的儿童特别容易感染，大约50%-90%的儿童死亡 在五岁之前。通过早期诊断和支持性护理，这种负担可以大大减轻 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。然而，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 由NIH/NHLBI资助的财团(SPARCO)在加纳、尼日利亚和坦桑尼亚设有协作站点 为未来在SCD方面的研究奠定基础。尼日利亚的Sparco工厂设在 阿布贾大学镰状细胞疾病研究和培训英才中心。在第一个 在颁奖阶段，我们建立了与临床和数据协调有关的管理结构 中心，并积极参与财团工作组的活动。我们获得并维护了道德规范 批准；在镰刀非洲数据协调中心的指导下建立了红帽数据库 (SADaCC)；并使用统一的病例报告表在19个地点成功招募了4687名患者 与尼日利亚镰刀细胞支持协会合作。(图)我们提供了以下方面的专业培训 对915名医务人员(包括初级卫生保健人员)的SCD研究和保健管理 中心(PHCS)。我们为临床研究硕士学位和研究生开发了课程 生物信息学文凭，以解决已发现的差距。我们分析了注册处的初步数据 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 在新生儿身上。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，以促进与SCD相关的流行病学、转化性和临床研究和政策 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队列增加到9,000人：a)额外招募2,000人，并额外引进6人 (B)扩大新生儿筛查，以建立1000名SCD患者的出生队列。我们会 扩展、实施和评估SPARCO护理标准(SOC)指南的后续使用情况 在SPARC-net网站上对SCD患者进行培训和管理，并确定培训的影响，以及 与卫生工作者、家长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，这些研究可能是 由财团采用，包括a)评估患者的特征和临床事件的发生率 Sparco SoC，b)评估HU使用对以下患者的临床和血液学结果的影响 SPARC-Net队列，以及c)使用基因组方法研究 SPARC-Net SCD出生队列中肠道微生物群、HbF水平和血管闭塞危象的频率。

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.