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: 10182610
• 负责人: Sarah Kiguli
• 金额: $ 10.72万
• 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/10182610
• 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; Enrollment; 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; Research Training; 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; fight against; graduate student; hydroxyurea; implementation research; implementation science; improved; innovation; mortality; 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%) 镰状细胞病 (SCD) 患者生活在撒哈拉以南非洲 (SSA)，那里 在高收入人群中，这种情况每年约占 5 岁以下儿童死亡率的 16%（15 万人死亡）。 国家采用基于证据的战略来控制 SCD 引起的死亡率和发病率，使 SCD 死亡率和发病率降低了 68% 1983 年至 2002 年 0-3 岁 SCD 儿童死亡率。这一成功是通过以下四项举措取得的 干预措施：通过积极预防，尽早发现 SCD； 致命并发症的检测和治疗，特别是感染；羟基脲作为疾病的用途 乌干达是总体护理水平较高的 SSA 国家之一。 镰状细胞 (HbS) 基因患病率达 13%，占乌干达儿童死亡率的 18%。 据该部门估计，50-80% 的患有这种疾病的儿童会在五岁生日之前死亡。 麦克雷雷大学健康科学学院儿科和儿童健康中心在 1968 年。诊所拥有镰状细胞数据库，其中包含超过 10,000 条独特的患者记录，并增加了 1000 条 该团队每年都会有新的注册病例，制定了当地的 SCD 患者管理指南， 培养学生，除了开展高质量的研究项目和 NIH 资助的研究之外。 然而，仍然存在的挑战包括： 1) 对所有患有此病的患者的综合护理不足 乌干达的研究记录了 SCD 相关发病率和死亡率的高负担。 中风、神经认知障碍、急性胸部综合征和微量白蛋白尿的患病率很高 2) 由于缺乏全面的新生儿筛查计划，许多儿童患有 SCD。 在晚年被诊断出或在诊断前死亡，以及 3) 羟基脲的使用率较低，尽管发现它 在这种情况下使用是安全有效的，麦克雷雷大学的团队正在申请加入 Sickle。 泛非研究联盟 (SPARCO) 作为合作联盟新站点之一，拥有 改善乌干达 SCD 疾病患者健康结果的目标，通过 我们应利用从中汲取的资源和经验教训。 NIH 在乌干达的投资包括 H3Africa 生物样本库（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) 为实现这一目标，将通过以下具体目标来实现： 目标 1： 在穆拉戈国家医院开发一个集中的、电子的、经患者同意的血红蛋白病登记系统 转诊镰状细胞诊所目标 2：通过建立 SCD 改善乌干达 SCD 患者的治疗效果。 目标 3：通过开展队列研究来推进 SCD 相关研究。 对 SCD 登记处登记的患者进行实施科学研究； 关于 SCD 青少年从儿科护理过渡到成人护理的另一项研究表明， 到本五年结束时，建立有效的协调和管理研究经费的机制。 支持，我们期望有一个系统来支持高质量 SCD 研究的可持续性 乌干达和 SSA 的临床护理可扩展。