Abstract
Background: Sickle cell disease (SCD) is one of the most common
inherited red blood cell disorders in the world, and the vast majority
of infants born with SCD are from Sub-Saharan Africa (SSA). Recent
studies have definitively established that hydroxyurea is safe and
highly effective for children with SCD in SSA. However, there is a lack
of data on the long-term outcomes for children with SCD in SSA beyond
early childhood. Procedure: We conducted a retrospective
analysis of AYA SCD patients ≥16 yo who received care in a pediatric SCD
clinic in Malawi between January 2015 and December 31, 2022
Results: We identified 207 patients ≥16 yo in the SCD clinic
electronic database. The vast majority (195, 94%) were alive and
receiving care in the pediatric SCD clinic. Three patients had died, and
9 were lost to follow-up. The mean age of patients alive and receiving
care was 18±1.9 yo. The mean age at diagnosis was 5.7±1.1 yo. Nearly all
AYA patients (N=187, 96%) were taking hydroxyurea for a mean duration
of 6±2.7 years. Mean baseline laboratory values were: hemoglobin 8.0±1.3
g/dL, mean corpuscular volume 92±8.4 fl, white blood count 14.2±7.0 × 10
3 /µL, absolute neutrophil count (ANC) 6.2±4.8 × 10
3 /µL, and platelets 343±167 × 10 3
/µL. Among all patients, 21% had at least one transfusion, and 23% of
patients had been hospitalized at least once for sickle cell-related
complications. Conclusion: We provide the first description of
an AYA cohort with SCD receiving routine care with hydroxyurea in a
dedicated pediatric clinic under real-world conditions. This study
highlights hydroxyurea’s long-term feasibility and underscores the need
to enhance the capacity for comprehensive adult care, especially in
SSA’s rapidly expanding AYA population.