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Hydroxyurea therapy in UK children with sickle cell anaemia: A single-centre experience

Kate Phillips1, Laura Healy2, Louise Smith2

  • 1Department of Women's and Children's Health, Institute of Translational Medicine, University of Liverpool, Liverpool, UK.

Pediatric Blood & Cancer
|October 9, 2017
PubMed

Insights

High-dose hydroxyurea is safe and effective for children with sickle cell anaemia, improving fetal hemoglobin levels and maintaining normal growth. This disease-modifying therapy is recommended for all pediatric patients.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacology

Background:

  • Hydroxyurea therapy is underutilized in UK pediatric sickle cell anaemia (SCA) patients, contrasting with global practices.
  • Concerns regarding toxicity, particularly marrow suppression and growth impairment, limit its use in the UK.
  • Current UK management often relies on supportive care or transfusions, reserving hydroxyurea for severe cases.

Purpose of the Study:

  • To evaluate the safety and efficacy of high-dose hydroxyurea in pediatric SCA patients in a UK setting.
  • To assess the impact of hydroxyurea dosage on fetal hemoglobin levels and hematological parameters.
  • To investigate potential toxicities, including marrow suppression and effects on growth, associated with hydroxyurea treatment.

Main Methods:

  • A cohort of 37 pediatric patients with SCA receiving hydroxyurea at a UK center was monitored.
  • Patients were analyzed based on hydroxyurea dosage: ≥26 mg/kg/day versus <26 mg/kg/day.
  • Hematological parameters, including fetal hemoglobin, mean cell volume, reticulocyte count, neutrophil count, and platelet count, were assessed.

Main Results:

  • Hydroxyurea therapy was well-tolerated, with mild transient cytopenias as the primary toxicity.
  • Higher doses (≥26 mg/kg/day) significantly increased fetal hemoglobin (Hb) levels (29.2% vs. 20.4%).
  • No significant marrow suppression or growth impairment was observed even with high-dose treatment; good adherence reduced hospitalizations.

Conclusions:

  • High-dose hydroxyurea is an effective and safe disease-modifying therapy for pediatric sickle cell anaemia.
  • The study supports the broader use of hydroxyurea in all children with SCA, irrespective of disease severity.
  • Findings challenge previous concerns about toxicity and advocate for hydroxyurea as a standard treatment option.
Abstract

Keywords:
hydroxyureapaediatricred blood cell disorderssickle cell disease

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