HYDROXYUREA IN PEDIATRIC PATIENTS WITH SICKLE CELL DISEASE

FACT SHEET

• Sickle cell anemia is a major public health problem affecting a significant portion of the African-American population.

• In 1995, an NHLBI clinical trial of hydroxyurea in adults over the age of 18 demonstrated, for the first time, an effective therapy for reducing painful episodes in severely affected adults with sickle cell anemia.

• Although hydroxyurea has been approved by the FDA for adults with sickle cell disease, there is concern about the potential toxicity of hydroxyurea in children, and its effects on growth and development.

• Therefore, hydroxyurea is not recommended for children at this time. However, clinicians are interested in determining its benefits, if any, in children particularly in preventing organ damage and improving long-term survival.

• A Phase I/II study ( PED-HUG) was supported by the NHLBI to determine the therapeutic dose of hydroxyurea in children with 3 or more painful episodes per year and to identify any short-term toxicities.

• The study was conducted in 4 of the 10 Comprehensive Sickle Cell Centers and 3 other clinical sites in children ages 5 to 15 years.

• The PED-HUG recently reported data on 50 children followed at the maximum tolerated dose of hydroxyurea for one year. There were no significant side effects with regard to growth and development or major organ dysfunction.

• Hydroxyurea was observed to increase fetal hemoglobin in a manner similar to that seen in the adults with sickle cell anemia, and is considered to be safe in children between the ages of 5 and 15 years.

• Further clinical research is needed in children to investigate the effectiveness of hydroxyurea in preventing long-term damage to major organs. This will be of great benefit if spleen function can be preserved, and the deleterious effects to the central nervous system, lungs and kidneys can be prevented.

June 1998

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