Sickle Pan-African Research Consortium (SPARCO)- Tanzania

项目总结 镰状细胞病(SCD)的负担在撒哈拉以南非洲(SSA)最高，那里75%以上的患者 目前居住在。坦桑尼亚是受该疾病影响最严重的国家之一，估计有11,000名新生儿 一年，在世界上排名第五。在SSA中解决SCD负担的主要挑战是 缺乏多地点、描述良好的SCD患者队列；未能部署一致的护理标准；人类 资源能力在数量和技能上有限，在与非洲可持续发展相关的领域研究有限 背景。2017年，坦桑尼亚、加纳和尼日利亚这三个SSA国家联手成立了Sickle 泛非研究联盟(SPARCO)应对这些挑战。以已取得的成功为基础 Date，Sparco-坦桑尼亚的目标是扩大成为SCD培训和研究的卓越中心 长期目标是推进SCD的护理，并为财团提供证据做出贡献 告知在SSA中部署有效的干预措施。具体地说，斯巴科-坦桑尼亚旨在1)加强 在SSA网络中与SCD成员进行沟通和协作，以参与整个大陆的 研究并创建一个“政策翻译小组”，将循证最佳做法嵌入国家 政策，2)通过以下方式提高SPARCO数据库中数据的数量和质量：i)增加 通过整合桑给巴尔的一个新卫星站点并实施新生儿，将SCD患者纳入7,000人 全国筛查(NBS)，(与美国血液学会合作)；ii)使用短信 提醒和针对特定年龄的患者教育计划，以提高门诊就诊率，在后续访问中收集 纵向数据；3)提高定制的多级SCD护理标准(SoC)在 坦桑尼亚卫生系统，与卫生部合作，促进采用和使用SOC 从三级护理级别到家庭；4)与财团内外的现有计划合作进行 SCD临床管理和研究的短期、中期和长期培训和奖学金计划 加强SCD护理和研究方面的技能；以及5)进行队列研究，以提高对 疾病和风险修饰物的现状(特别是关于胎儿血红蛋白和 肾功能障碍和羟基脲对免疫激活的影响)，以及评估的实施研究 计划的干预措施(特别是国家BS的可行性、可接受性和可持续性、羟基尿素的使用 和疟疾预防)，旨在阐明扩大采用这些预防和预防措施的最佳方法 在当地背景下的治疗干预措施。总体而言，该项目将提高 SSA中关于SCD的数据，使研究人员的数量增长到临界值，并生成将有助于 政策变化，与护理标准的增长同时，将导致减少 SSA中SCD的发病率和死亡率。

PROJECT SUMMARY The burden of sickle cell disease (SCD) is highest in sub-Saharan Africa (SSA) where over 75% of patients currently reside. Tanzania is one of the countries most affected by the disease with an estimated 11,000 births a year, the 5th highest in the world. Major challenges in addressing the burden of SCD in SSA have been the lack of multi-site, well described cohorts of SCD patients; failure to deploy consistent standards of care; human resource capacity that is limited in number and skills and limited research in areas pertinent to SCD in the African context. In 2017 three SSA countries, Tanzania, Ghana and Nigeria, teamed up to establish the Sickle Pan-African Research Consortium (SPARCO) to address these challenges. Building on successes achieved to date, SPARCO-Tanzania aims to expand as the Center of Excellence in Training and Research in SCD with the long term goal to advance the care for SCD and contribute to the consortium in generating evidence that will inform the deployment of effective interventions in SSA. Specifically, SPARCO-Tanzania aims to 1) Enhance communication and collaboration with members of the SCD in SSA Network to engage in continent-wide research and create a “Policy Translation Group” that will embed evidence-based best practices in national policy, 2) Increase the quantity and quality of data in the SPARCO database by i) increasing the number of enrolled SCD patients to 7,000 by integrating a new satellite site in Zanzibar and implementing newborn screening (NBS) across the country, (in collaboration with the America Society of Hematology); ii) using SMS reminders and age-specific patient educational programs to boost clinic attendance at follow-up visits to collect longitudinal data; 3) Enhance the utilization of the customized multi-level SCD Standards of Care (SoC) in the Tanzanian health system by engaging with the Ministry of Health to promote the adoption and use of the SoC from tertiary care level to home; 4) partner with existing programs in and outside the consortium to conduct short, medium and long term training and fellowship programs on the clinical management and research on SCD to strengthen skills in SCD care and research; and 5) Conduct cohort studies to improve understanding of current status of the disease and risk-modifiers (specifically on the genetic variability in fetal hemoglobin and kidney dysfunction and effect of Hydroxyurea on immune activation), and implementation research to evaluate planned interventions (specifically the feasibility, acceptability and sustainability of NBS, Hydroxyurea usage and malaria prophylaxis) aiming at elucidating best approaches to scale-up introduction of these preventive and therapeutic interventions in the local context. Collectively, the project will increase the amount and quality of data on SCD in SSA, enable growth of a critical mass of researchers and generate evidence that will inform policy change which, concurrent with the growth in the standards of care, will lead to the reduction in the morbidity and mortality due to SCD in SSA.