title: Hydroxyurea for children with sickle cell disease.
abstract: 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.
authors.affiliation: Harvard Medical School, Boston, MA, USA. matthew.heeney@childrens.harvard.edu
authors.forename: Matthew M
authors.initials: MM
authors.lastname: Heeney
authors.forename: Russell E
authors.initials: RE
authors.lastname: Ware
mesh_terms: D000755:Anemia, Sickle Cell / Q000188:drug therapy*; D000986:Antisickling Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage / Q000627:therapeutic use*
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea