Hydroxyurea therapy for sickle cell anemia

Expert Opin Drug Saf. 2015;14(11):1749-58. doi: 10.1517/14740338.2015.1088827. Epub 2015 Sep 14.


Introduction: Sickle cell anemia (SCA) is a severe, inherited hemoglobin disorder affecting 100,000 persons in the US and millions worldwide. Hydroxyurea, a once daily oral medication, has emerged as the primary disease-modifying therapy for SCA. The accumulated body of evidence over 30 years demonstrates that hydroxyurea is a safe and effective therapy for SCA, but hydroxyurea remains underutilized for a variety of reasons.

Areas covered: In this review, we summarize the available evidence regarding the pharmacology, clinical, and laboratory benefits, and safety of hydroxyurea therapy for the treatment of SCA. The purpose of this review is to provide the reader a comprehensive understanding of hydroxyurea and to reinforce the fact that hydroxyurea is a safe and effective medication for the treatment of SCA.

Expert opinion: In our opinion, hydroxyurea therapy should be considered standard-of-care for SCA, representing an essential component of patient management. Early initiation and broader use of hydroxyurea will alter the natural history of SCA, so affected children can live longer and healthier lives. In addition, hydroxyurea use should be extended to low-resource settings such as sub-Saharan Africa, where the burden of SCA and the need for hydroxyurea is arguably the greatest.

Keywords: fetal hemoglobin; hemoglobinopathies; hydroxyurea; sickle cell anemia.

Publication types

  • Review

MeSH terms

  • Administration, Oral
  • Anemia, Sickle Cell / drug therapy*
  • Animals
  • Antisickling Agents / administration & dosage
  • Antisickling Agents / adverse effects
  • Antisickling Agents / therapeutic use*
  • Humans
  • Hydroxyurea / administration & dosage
  • Hydroxyurea / adverse effects
  • Hydroxyurea / therapeutic use*


  • Antisickling Agents
  • Hydroxyurea