Improving Sickle Cell Disease Care in Mali through Engagement with a Pan-African Sickle Cell Disease Network.

通过与泛非镰状细胞病网络的合作，改善马里的镰状细胞病护理。

基本信息

批准号：
10625502
负责人：
ALDIOUMA GUINDO
金额：
$ 15.81万
依托单位：
UNIV OF SCIENCES, TECH & TECH OF BAMAKO
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-05-11 至 2026-04-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10625502
关键词：
Acceleration Acute Address Africa Africa South of the Sahara African Age Antibiotics Antimalarials Bacterial Infections Caring Cephalic Chemoprophylaxis Child Clinical Clinical Data Clinical Protocols Communicable Diseases Communities Counseling Data Data Collection Development Diagnosis Diagnostic Services Education Educational Intervention Electronics Enrollment Ensure Etiology Evidence based intervention Face Family Fetal Hemoglobin Fever Fingers Future Geography Goals Health Health Personnel Heart Diseases Hematological Disease Heterozygote Incidence Individual Infection Inherited Institutional Review Boards Kidney Diseases Life Lifting Longitudinal cohort Longitudinal cohort study Malaria Mali Measures Methods Molecular Diagnosis National Heart, Lung, and Blood Institute Neonatal Screening Observation in research Observational Study Ontology Organ Outcome Outpatients Pain Pain management Patients Persons Population Predisposition Preventive Preventive care Process Prophylactic treatment Protocols documentation Proverb Quality of Care Registries Research Risk Reduction Safety Services Sickle Cell Sickle Cell Anemia Sickle Cell Trait Site Standardization Stroke Teleconferences Training Transfusion Vaccination Vaccines Vascular Diseases Visit acute care aged cardiovascular disorder risk clinical care clinical practice cohort cost effectiveness high risk hydroxyurea implementation determinants implementation evaluation implementation outcomes implementation research improved infection risk instrument meetings participant enrollment patient population quality assurance research study response risk mitigation sample collection screening screening program sickling standardized care stroke risk treatment center

项目摘要

PROJECT SUMMARY Sickle cell disease (SCD) is an inherited blood disease that causes pain, susceptibility to infection, and greatly increases the risk of cardiovascular diseases such as stroke, kidney disease, and heart disease. SCD is common in Africa because individuals with sickle cell trait (i.e., heterozygotes) are protected against severe malaria. Unfortunately, there are now more than 235,000 children born with SCD in sub-Saharan Africa each year. Undiagnosed, 50-90% of these children could die before the age of five years, mostly from bacterial infections to which children with SCD are highly susceptible. Newborn screening programs across Africa need to be expanded towards universal screening for SCD so that children born with this condition can receive evidence-based interventions to manage pain, reduce the risk of infections, and slow the progression of vascular diseases. To address these challenges, a pan-African sickle cell network has been established and is supported by the NHLBI. In this proposal, we introduce a busy sickle cell treatment center based in Bamako, Mali and explain how this center establish a registry of 4,000 patients with SCD utilizing electronic clinical data capture, deliver consistent Standards of Care with ongoing quality assurance and quality improvement, and conduct observational and implementation research to address the most pressing questions facing African people with SCD. The Centre Recherche et de Lutte contre la Drepanocytose (CRLD) is comprehensive sickle cell care center established in 2010 that now serves more than 12,000 people with SCD. We continue to enroll 120-150 new patients each month. We provide diagnostic services, counseling and education, as well as preventive and acute care for people with SCD. To establish a longitudinal cohort of 4,000 patients with SCD, we will develop data capture instruments in coordination with the Consortium, establish new IRB-approved protocols, and begin enrolling patients at a rate of 500 in the first year and 1,000 each subsequent year. The descriptive clinical data and biospecimens collected on enrollment will be shared with the Consortium. We propose delivering Standards of Care that address the most pressing needs of the SCD community. These include (I) providing diagnosis, counseling, and education; (II) mitigating the risk of infectious disease by providing vaccines, antibiotics and malaria chemoprophylaxis; and (III) providing hydroxyurea, an inducer of fetal hemoglobin expression and a proven therapy for SCD, to high-risk patients. Finally, we propose observational and implementation research studies that will lead to improved care of people with SCD. These include (I) studying the impact of our counseling and education interventions; (II) describing the etiologies and consequences of fever in the outpatient setting; and (III) extending malaria chemoprophylaxis to older children. Together, we aim to improve the care and treatment of African people with SCD both now and in the future.

项目概要镰状细胞病 (SCD) 是一种遗传性血液疾病，会导致疼痛、易受感染，并极大地影响患者的健康。增加患中风、肾病和心脏病等心血管疾病的风险。 SCD 是在非洲很常见，因为具有镰状细胞特征的个体（即杂合子）可以免受严重的侵害疟疾。不幸的是，目前撒哈拉以南非洲地区有超过 235,000 名出生时患有 SCD 的儿童年。如果未经诊断，这些儿童中的 50-90% 可能会在五岁之前死亡，大部分死于细菌感染患有 SCD 的儿童极易受到感染。整个非洲的新生儿筛查项目需要扩大对 SCD 的普遍筛查，以便出生时患有这种疾病的儿童能够接受基于证据的干预措施来控制疼痛、降低感染风险并减缓病情进展血管疾病。为了应对这些挑战，泛非洲镰状细胞网络已经建立并正在实施中得到 NHLBI 的支持。在本提案中，我们介绍了位于巴马科的繁忙镰状细胞治疗中心，马里并解释该中心如何利用电子临床数据建立 4,000 名 SCD 患者的登记册捕获、提供一致的护理标准以及持续的质量保证和质量改进，以及进行观察和实施研究，以解决非洲面临的最紧迫问题患有 SCD 的人。 Center Recherche et de Lutte contre la Drepanocytose (CRLD) 是综合性镰状细胞护理中心成立于 2010 年，目前为超过 12,000 名 SCD 患者提供服务。我们继续招收120-150名新人每个月都有病人。我们提供诊断服务、咨询和教育以及预防和治疗 SCD 患者的紧急护理。为了建立 4,000 名 SCD 患者的纵向队列，我们将开发数据采集仪器与联盟协调，制定新的 IRB 批准的方案，并开始按一定比例招募患者第一年 500 名，随后每年 1,000 名。描述性临床数据和生物样本注册时收集的信息将与联盟共享。我们建议提供满足 SCD 社区最紧迫需求的护理标准。其中包括 (I) 提供诊断、咨询和教育；（二）降低传染病风险提供疫苗、抗生素和疟疾化学预防； (III)提供羟基脲，其诱导剂针对高危患者的胎儿血红蛋白表达和经证实的 SCD 治疗方法。最后，我们提出观察性和实施性研究，这将改善对患者的护理患有 SCD 的人。这些包括（I）研究我们的咨询和教育干预措施的影响；（二）描述门诊发热的病因和后果； (III) 疟疾蔓延对年龄较大的儿童进行化学预防。我们共同致力于改善现在和未来非洲 SCD 患者的护理和治疗。