Hydroxyurea in children with sickle cell disease in a resource-poor
setting: Monitoring and effects of therapy. A practical perspective
Abstract
Abstract Background: Effectiveness of hydroxyurea (HU) in SCD is well
established. Unanswered questions persist about use in African children
in terms of acceptability and monitoring. We determined real-life
user-barriers, safety and effects of therapy among children using HU in
Nigeria. Methods: We retrieved and reviewed case notes of children on
hydroxyurea (10-15mg/kg/day followed by dose escalation) from January
2017 to June 2019, checked for adherence to drugs, clinics and
laboratory tests; starting dose, toxicity and benefits (hematologic,
clinical, parental satisfaction). A questionnaire complemented case note
findings. Results 116 patients received hydroxyurea (mean dose of 18
mg/kg/ day). Improvement in general well-being was 91.25%, reduction in
bone pain 83.3%, hospital admissions 71.9%, abdominal pain 62.3%,
blood transfusion 56.1%. Sixteen percent voluntarily stopped HU because
of cost and side effects. Sixty-seven percent of parents complained
about daily drug use, frequency and cost of monitoring. Adherence to
daily HU was 88.8%, doctor’s appointments 22.8%, hematology tests
17.5%, organ function 32.5%. Five patients had mild neutropenia. No
significant hepatic or renal toxicity occurred. Common side effects were
abdominal pain (18.4%) and headache (13.2%). Significant increase in
hemoglobin, fetal hemoglobin, MCV and reduction in absolute neutrophil
counts occurred. Conclusion: Hydroxyurea at 10-15mg/kg/day is safe,
effective and acceptable to parents. Monitoring is challenging.
Care-giver education, support services, staff training, HU clinics may
improve utilization. HU protocols that reduce monitoring and yet provide
good clinical and laboratory benefits are necessary. This study adds to
the implementation science strategies that is needed to increase HU use
in Nigeria.