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.
