The U.S. Food and Drug Administration (FDA) approved hydroxyurea tablets to reduce pain crises in pediatric patients with sickle cell anemia. The therapy is the first and only hydroxyurea-based treatment for this indication.
The approval was based on safety and efficacy data from the European Sickle Cell Disease Cohort study (ESCORT-HU) of 405 pediatric patients with sickle cell disease (SCD) ages 2 to 18. For the 141 patients eligible for analysis, hydroxyurea use resulted in an increase in hemoglobin F. After 12 months of treatment, the drug also decreased the percentage of patients who experienced at least one vaso-occlusive episode, acute chest syndrome episode, hospitalization due to SCD, or blood transfusion.
The most common adverse reactions (incidence >10%) included infections and neutropenia. The recommended initial dose of hydroxyurea is 20 mg/kg once-daily.
Sources: Rare Disease Report, March 15, 2018; European Society for Medical Oncology press release, January 5, 2018.