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Hematol Oncol Clin North Am. 2010 Feb;24(1):199-214. doi: 10.1016/j.hoc.2009.11.002.

Hydroxyurea for children with sickle cell disease.

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  • 1Department of Pediatrics, Harvard Medical School, Children's Hospital Boston, 300 Longwood Avenue, Boston, MA 02115, USA. matthew.heeney@childrens.harvard.edu

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.

Copyright (c) 2010 Elsevier Inc. All rights reserved.

PMID:
20113903
[PubMed - indexed for MEDLINE]
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