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Hydroxyurea in children with sickle cell disease in a resource-poor setting: Monitoring and effects of therapy. A

Uche Nnebe-Agumadu1, Innocent Adebayo2, Ifeanyi Erigbuem2

  • 1Department of Paediatrics, College of Health Sciences, University of Abuja, Abuja, Nigeria.

Pediatric Blood & Cancer
|March 31, 2021
PubMed

Insights

Hydroxyurea (HU) is safe and effective for Nigerian children with sickle cell disease, improving well-being and reducing complications. Challenges include daily adherence and monitoring costs, necessitating improved access and support.

Area of Science:

  • Hematology
  • Pediatrics
  • Pharmacology

Background:

  • Effectiveness of hydroxyurea (HU) for sickle cell disease (SCD) is known, but its use in African children requires further investigation.
  • Unanswered questions persist regarding the real-world acceptability, availability, and monitoring of HU in Nigeria.

Purpose of the Study:

  • To determine real-life issues of acceptability, availability, and monitoring of hydroxyurea (HU) use in Nigerian children with sickle cell disease.
  • To assess the safety, efficacy, and parental perception of HU therapy in a pediatric SCD population in Nigeria.

Main Methods:

  • Retrospective longitudinal review of laboratory data from patient case files.
  • Cross-sectional survey assessing families' perceptions of medication adherence, clinic visits, laboratory tests, benefits, and side effects.

Main Results:

  • 116 pediatric patients received HU; 89 had lab analysis. Dose escalation occurred in 80%. Parents reported improved well-being, reduced pain, hospitalizations, and transfusions.
  • 89.5% of parents were satisfied with HU, but 61% were dissatisfied with daily dosing and monitoring frequency/cost. Adherence varied: 88.8% for daily HU, 24.5% for appointments, 18.9% for hematology tests, 37.4% for organ function tests.
  • Despite inconsistent dose escalation, significant improvements in hemoglobin, hemoglobin F, mean corpuscular volume, and reduction in absolute neutrophil count were observed with no significant toxicities.

Conclusions:

  • Hydroxyurea (HU), with a starting dose of 10-15 mg/kg/day, is safe, effective, and acceptable for Nigerian children with sickle cell disease.
  • Key considerations for initiating HU therapy include parental education, affordable drug provision, and subsidized laboratory tests.
  • Specialized HU clinics could improve dose escalation and monitoring; further studies on maximum tolerable dose protocols in sub-Saharan Africa are needed.
Abstract

Keywords:
hydroxyureamonitoringresource-poor settingsafetysickle cell disease

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