Hydroxyurea therapy in children severely affected with sickle cell disease.
Hydroxyurea is an effective drug for the treatment of severe sickle cell disease (SCD) in children.
The major clinical manifestation of SCD is haemolytic anaemia, predisposition to infection and recurrent vaso-occlusive episodes resulting in pain, organ dysfunction, or both. There has been no satisfactory treatment for children with recurrent severe painful episodes caused by SCD.
Hydroxyurea treatment of severe SCD in children is certainly confirmed to be associated with improved haematological parameters, acceptable toxic effects and a trend to reduced hospitalization.
Children with sickle cell anaemia provide the best opportunity to assess the efficacy of hydroxyurea (HU) in preventing complications and progressive organ damage. Hydroxyurea is an antimetabolite drug shown to increase fetal haemoglobin levels and reduce the symptoms of SCD.
A prospective, controlled trial to investigate the efficacy of Hydroxyurea in children is therefore warranted.