Sickle Pan-African Research Consortium (SPARCO) Uganda: Strengthening Capacity for Clinical Care, Research and Training in Sickle Cell Disease - SCRT Project.

镰状细胞泛非研究联盟 (SPARCO) 乌干达：加强镰状细胞病临床护理、研究和培训的能力 - SCRT 项目。

• 批准号: 10634627
• 负责人: Sarah Kiguli
• 金额: $ 16.19万
• 依托单位: MAKERERE UNIVERSITY COLLEGE OF HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-06-01 至 2026-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10634627
• 关键词: Acute; Address; Adolescent; Adopted; Adoption; Adult; Affect; Africa South of the Sahara; African; Age; Anemia; Area; Caring; Cessation of life; Characteristics; Child; Child Health; Child Mortality; Childhood; Clinic; Cohort Studies; Comprehensive Health Care; Consent; Country; Data; Databases; Detection; Diagnosis; Disease; Disease Management; Disease Outcome; Electronics; Erythrocytes; Ethical Issues; Ethics; Evidence based intervention; Formulation; Funding; Genes; Genetic; Genetic Counseling; Goals; Grant; Guidelines; Health; Health Sciences; Hematological Disease; Hematology; Hemoglobin; Hereditary Disease; High Prevalence; Income; Individual; Infection; Intervention; Investments; Legal; Life; Maintenance; Medical; Microalbuminuria; Morbidity - disease rate; Natural History; Neonatal Screening; Neurocognitive Deficit; Oncology; Outcome; Pain; Participant; Patient-Focused Outcomes; Patients; Penicillins; Phenotype; Policies; Prevalence; Prevention; Preventive; Prophylactic treatment; Recording of previous events; Records; Recurrence; Registries; Research; Research Project Grants; Resources; Rest; SERPINI2 gene; Sickle Cell; Sickle Cell Anemia; Site; Stroke; System; Time; Training; Uganda; United States National Institutes of Health; Universities; Work; acute chest syndrome; biobank; burden of illness; chemotherapy; clinical care; clinical phenotype; cohort; college; data management; disease registry; electronic registry; evidence base; fighting; graduate student; hydroxyurea; implementation research; implementation science; improved; innovation; mortality; participant enrollment; pediatric department; programs; recruit; research study; screening; screening program; sickling; student training; success; trait

Project Abstract: Most (75%), individuals living with Sickle Cell Disease (SCD) are found in sub-Saharan Africa (SSA), where the condition accounts for about 16% (150,000 deaths) annually of under-5 mortality. In high income countries, evidence-based strategies to control mortality and morbidity due to SCD, reduced by 68% the mortality of children 0 – 3 years with SCD from 1983 and 2002. This success was achieved through four interventions: Detection of SCD as early as possible in life, through screening; aggressive prevention, detection and treatment of fatal complications, especially infections; The use of hydroxyurea as a disease- modifying agent; comprehensive care programs. Uganda is one of the SSA countries with a high overall sickle cell (HbS) gene prevalence of 13%. SCD accounts for 18% of child mortality in Uganda with estimates that 50-80% of children with the condition die before their fifth birthday. The Department of Pediatrics & Child Health, Makerere University College of Health Sciences established a sickle cell clinic in 1968. The clinic has a sickle cell database with over 10,000 unique patient records that increases by 1000 new registrations annually. The team has developed local guidelines for management of patients with SCD, trains students, in addition to conducting high-quality research projects and NIH funded studies. There are however remaining challenges that include: 1) Inadequate comprehensive care to all patients that has resulted in high burden of SCD-related morbidity and mortality. Research done in Uganda has documented high prevalence of stroke, neurocognitive impairments, acute chest syndrome, and micro-albuminuria among children with SCD. 2) due to a lack of a comprehensive newborn screening program, many children are diagnosed later in life or die before diagnosis, and 3) Low use of hydroxyurea despite the findings that it is safe and effective for use in this setting. The team at Makerere University is applying to join the Sickle Pan-African Research Consortium (SPARCO) as one of the Collaborative Consortium new sites, with a goal of improving the health outcomes of individuals with of SCD disease in Uganda, through implementation of evidence-based interventions. We shall leverage the resources and lessons learnt from NIH investments in Uganda including the H3Africa biorepository (UH2 HG007051; Joloba), MEPI project (R24TW008886, Sewankambo) and other ongoing grants including the HEPI project (1R25TW011213-01, Kiguli) to achieve this. The project goal will be achieved through the following specific aims: Aim 1: To develop a centralized, electronic, patient consented haemoglobinopathy registry at Mulago National Referral Sickle Cell Clinic. Aim 2: To improve SCD patient outcomes in Uganda by establishing SCD standards of care. Aim 3: To advance SCD related research by conducting cohort studies and implementation science studies on patients enrolled in the SCD registry; and an additional study on additional study on the transition from pediatric to adult care by SCD adolescents. MakCHS has well- established, efficient mechanisms for coordinating and managing research grants. By the end of this 5-year support, we anticipate having a system to support the sustainability of high quality SCD research and clinical care that is scalable in Uganda and SSA.

项目摘要： 大多数（75％），患有镰状细胞病（SCD）的人都在撒哈拉以南非洲（SSA）中发现 该病每年约为5岁以下死亡率的16％（150,000人死亡）。高收入 国家，基于证据的策略来控制SCD引起的死亡率和发病率，减少了68％ 从1983年和2002年开始使用SCD的儿童死亡率0 - 3年。这一成功是通过四个 干预措施：通过筛查尽早发现SCD；积极的预防， 检测和治疗致命并发症，尤其是感染；使用羟基脲作为疾病 - 修改代理；综合护理计划。乌干达是SSA国家之一 镰状细胞（HBS）基因患病率为13％。 SCD占乌干达儿童死亡率的18％ 估计有50-80％的病情在五岁生日之前死亡。部门 Makerere大学健康科学学院儿科与儿童健康建立了一家镰状细胞诊所 1968年。诊所有一个镰状细胞数据库，有超过10,000个独特的患者记录，增加了1000个 每年新注册。该团队已制定了当地的SCD患者管理指南， 培训学生，除了进行高质量的研究项目和NIH资助的研究外。有 但是剩下的挑战包括：1）所有患者的全面护理不足 导致与SCD相关的发病率和死亡率负担很高。在乌干达进行的研究已记录 中风，神经认知障碍，急性胸部综合征和微藻尿症的高流行率 在患有SCD的孩子中。 2）由于缺乏全面的新生儿筛查计划，许多孩子 在诊断之前诊断或在诊断前死亡，3）羟基脲目的地的使用率很低 在此环境中安全有效。 Makerere University的团队申请加入镰刀 泛非研究财团（SPARCO）是合作联盟的新网站之一， 通过 实施基于证据的干预措施。我们将利用从中学到的资源和教训 NIH在乌干达的投资包括H3africa Biorepository（UH2 HG007051; Joloba），MEPI项目 （R24TW008886，Sewankambo）和其他正在进行的赠款，包括HEPI项目（1R25TW011213-01， kiguli）实现这一目标。项目目标将通过以下特定目标实现：目标1： 在Mulago National开发集中式，电子，患者同意的血红蛋白病注册表 推荐镰状细胞诊所。目标2：通过建立SCD来改善乌干达的SCD患者结果 护理标准。目标3：通过进行队列研究和 关于参加SCD注册中心的患者的实施科学研究；还有一项关于 SCD青少年从小儿到成人护理过渡的其他研究。 Makchs有很好的 建立，有效的协调和管理研究补助机制。到这个五年结束时 支持，我们预计有一个系统来支持高质量SCD研究的可持续性和 乌干达和SSA可扩展的临床护理。