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