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Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell

Sherri A Zimmerman1, William H Schultz, Jacqueline S Davis

  • 1Duke Pediatric Sickle Cell Program and Division of Pediatric Hematology/Oncology, Duke University Medical Center, PO Box 2916, Durham, NC 27710, USA. zimme008@mc.duke.edu

Blood
|November 25, 2003
PubMed

Insights

Long-term hydroxyurea therapy is safe and effective for children with sickle cell disease (SCD). It improves blood counts and fetal hemoglobin (HbF) levels without significant side effects or impacting growth.

Area of Science:

  • Pediatric Hematology
  • Sickle Cell Disease Research
  • Pharmacological Interventions

Background:

  • Hydroxyurea is known to improve hematologic parameters in pediatric sickle cell disease (SCD).
  • However, the long-term efficacy and safety of hydroxyurea at its maximum tolerated dose (MTD) in this population remained undetermined.

Purpose of the Study:

  • To evaluate the long-term efficacy and safety of hydroxyurea therapy at MTD in pediatric patients with SCD.
  • To assess hematologic parameter changes and potential adverse effects over an extended treatment period.

Main Methods:

  • A cohort of 122 pediatric patients with SCD initiated hydroxyurea therapy between 1995 and 2002.
  • Therapy was escalated to MTD (average 25.4 mg/kg/day), with treatment durations averaging 45 months (range 6-101 months).
  • Hematologic parameters, fetal hemoglobin (HbF) levels, and adverse events were monitored.

Main Results:

  • Hydroxyurea therapy significantly increased hemoglobin and HbF levels while decreasing reticulocyte, white blood cell, and platelet counts.
  • Patients with various SCD genotypes showed hematologic responses.
  • Sustained HbF induction for up to 8 years was observed without adverse effects on growth or increased DNA mutations.

Conclusions:

  • Long-term hydroxyurea therapy at MTD is well-tolerated in pediatric SCD patients.
  • The treatment demonstrates sustained hematologic efficacy and apparent long-term safety.
  • Hydroxyurea is a viable therapeutic option for managing pediatric sickle cell disease.

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