Hydroxyurea for children with sickle cell disease

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


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / drug therapy*
  • Child
  • Humans
  • Hydroxyurea / adverse effects
  • Hydroxyurea / therapeutic use*
  • Maximum Tolerated Dose
  • Risk Assessment


  • Hydroxyurea