Hydroxyurea has been approved for use in pediatric sickle cell patients aged 2 years and older to reduce the frequency of painful crises and the need for blood transfusions, the Food and Drug Administration announced on Dec. 21.
This is the first FDA approval of hydroxyurea for use in pediatric sickle cell patients. The recommended initial dose is 20 mg/kg once daily but can be changed based on blood count levels, the agency said in a press release .
The approval of hydroxyurea was based on results from the European Sickle Cell Disease Cohort–Hydroxyurea study ( ESCORT UH ), an open-label, single-arm trial of 405 pediatric sickle cell patients between the ages of 2 and 18 years. Prior to enrolling in the study, 141 of the patients had not previously used hydroxyurea. Further study of the 141 patients found that hydroxyurea use led to an increase in hemoglobin F. After 12 months of hydroxyurea treatment, the percentage of patients who experienced vaso-occlusive episodes, acute chest syndrome, hospitalization due to sickle cell disease, or blood transfusions decreased.
The most common adverse reactions to hydroxyurea, infections and neutropenia, occurred in less than 10% of patients. Hydroxyurea causes severe myelosuppression and should not be administered to patients with depressed bone marrow function.
Hydroxyurea is manufactured as Siklos by Addmedica. More information concerning hydroxyurea indications, dosing, and precautions can be found here .
SOURCE: FDA press release .
