Hydroxyurea for children with sickle cell disease.
Hydroxyurea therapy offers promise for ameliorating the clinical course of children with sickle cell disease (SCD). Hydroxyurea is a prototypic therapeutic option; it can be administered with minimal side effects, has a relatively wide therapeutic window, and has mechanisms of action that address pathophysiologic pathways of sickling, vaso-occlusion, hemolysis, and organ damage. There are limited data regarding hydroxyurea's ability to prevent or diminish organ dysfunction, and the long-term risks of hydroxyurea therapy remain incompletely defined. Although clinical trials are underway to address long-term issues, hydroxyurea remains an effective but underutilized therapy for SCD.
Heeney
Matthew M
MM
Department of Pediatrics, Harvard Medical School, Children's Hospital Boston, 300 Longwood Avenue, Boston, MA 02115, USA. matthew.heeney@childrens.harvard.edu
Ware
Russell E
RE
D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy*; D002648:Child; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use*; D020714:Maximum Tolerated Dose; D018570:Risk Assessment
D006918:Hydroxyurea