Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2017 Feb 15;9(1):e2017015.
doi: 10.4084/MJHID.2017.015. eCollection 2017.

Hydroxycarbamine: from an Old Drug Used in Malignant Hemopathies to a Current Standard in Sickle Cell Disease

Giovanna Cannas  1 , Solène Poutrel  2 , Xavier Thomas  3
Affiliations
Review

Hydroxycarbamine: from an Old Drug Used in Malignant Hemopathies to a Current Standard in Sickle Cell Disease

Giovanna Cannas et al. Mediterr J Hematol Infect Dis. .

Abstract

While hydroxycarbamide (hydroxyurea, HU) has less and fewer indications in malignant hemopathies, it represents the only widely used drug which modifies sickle cell disease pathogenesis. Clinical experience with HU for patients with sickle cell disease has been accumulated over the past 25 years in Western countries. The review of the literature provides increasing support for safety and efficacy in both children and adults for reducing acute vaso-occlusive events including pain episodes and acute chest syndrome. No increased incidence of leukemia and teratogenicity was demonstrated. HU has become the standard-of-care for sickle cell anemia but remains underused. Barriers to its use should be identified and overcome.

Keywords: Clinical Management; Hemoglobinopathies; Hydroxyurea; Prognosis; Sickle Cell Anemia; Treatment.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Structure of hydroxycarbamine (hydroxyurea, HU).
See this image and copyright information in PMC

References

    1. Dresler WFC, Stein R. Über den hydroxylharnstoff. Justus Liebigs Ann Chem. 1869;150:242–52. https://doi.org/10.1002/jlac.18691500212. - DOI
    1. Rosenthal F, Wislicki L, Koller L. Über die beziehungen von schwersten blutgiften zu abbauprodukten des eiweisses: ein beitrag zum entstehungmechanismus der pernizosen anemie. Klin Wochenschr. 1928;7:972–7. https://doi.org/10.1007/BF01716922. - DOI
    1. Stearns B, Losee KA, Bernstein J. Hydroxyurea: a new type of potential antitumor agent. J Med Chem. 1963;6:201. https://doi.org/10.1021/jm00338a026. - DOI - PubMed
    1. Fishbein WN, Carbone PP, Freireich EJ, et al. Clinical trials of hydroxyurea in patients with cancer and leukemia. Clin Pharmacol Ther. 1964;5:574–80. https://doi.org/10.1002/cpt196455574. - DOI - PubMed
    1. Becloff GL. Pharmacological, metabolic and clinical experience with hydroxyurea. Clin Trials J. 1967;4:873–83.