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 pa...

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Bibliographic Details
Published inHematology/oncology clinics of North America Vol. 24; no. 1; p. 199
Main Authors Heeney, Matthew M, Ware, Russell E
Format Journal Article
LanguageEnglish
Published United States 01.02.2010
Subjects
Anemia, Sickle Cell - complications
Anemia, Sickle Cell - drug therapy
Child
Humans
Hydroxyurea - adverse effects
Hydroxyurea - therapeutic use
Maximum Tolerated Dose
Risk Assessment
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Summary: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.
ISSN:1558-1977
DOI:10.1016/j.hoc.2009.11.002