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A two-year pilot trial of hydroxyurea in very young children with sickle-cell anemia

W C Wang1, L W Wynn, Z R Rogers

  • 1Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

The Journal of Pediatrics
|December 18, 2001
PubMed

Insights

Hydroxyurea is feasible and well-tolerated in infants with sickle cell anemia (SCA), showing hematologic benefits and potentially delaying spleen dysfunction. Further research is needed to evaluate its organ-protective effects.

Area of Science:

  • Pediatrics
  • Hematology
  • Pharmacology

Background:

  • Sickle cell anemia (SCA) often leads to chronic organ damage in infants.
  • Hydroxyurea is effective in older patients but its use in infants is less understood.
  • Early intervention may prevent long-term complications.

Purpose of the Study:

  • To assess the feasibility and toxicity of hydroxyurea in infants with SCA.
  • To evaluate the hematologic effects of hydroxyurea in this population.
  • To determine the impact of hydroxyurea on spleen function in infants with SCA.

Main Methods:

  • A pilot trial involving 28 infants (median age 15 months) with SCA (Hb SS or Sbeta(0) thalassemia).
  • Patients received hydroxyurea at 20 mg/kg/day for 2 years, with temporary discontinuation for toxicity.
  • Feasibility, toxicity, hematologic changes, and spleen function (via radionuclide uptake) were assessed.

Main Results:

  • Hydroxyurea was feasible and generally well-tolerated, with transient neutropenia as the main toxicity.
  • After 2 years, mean Hb level was 8.8 g/dL and Hb F was 20.3%, exceeding predicted levels.
  • 47% of patients showed absent splenic uptake, suggesting preserved spleen function compared to the expected 80% asplenia.

Conclusions:

  • Hydroxyurea therapy is feasible and well-tolerated in infants with SCA.
  • The treatment demonstrated hematologic efficacy and may help preserve spleen function.
  • Further studies are warranted to confirm hydroxyurea's potential for organ protection in SCA infants.
Abstract

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