Role of hydroxycarbamide in prevention of complications in patients with sickle cell disease

le of hydroxycarbamide in prevention of complications in patients with sickle cell disease Review (3970) Total Article Views Authors: NM Wiles, J Howard Published Date September 2009 Volume 2009:5 Pages 745 - 755 DOI: http://dx.doi.org/10.2147/TCRM.S4769 NM Wiles, J Howard Department of Haematology, St Thomas’ Hospital, Westminster, Bridge Road, London, SE1 7EH, UK Abstract: Sickle cell disease (SCD) is a genetically inherited condition caused by a point mutation in the beta globin gene. This results in the production of the abnormal hemoglobin, sickle hemoglobin (HbS). Hydroxycarbamide, is an antimetabolite/cytotoxic which works by inhibiting ribonucleotide reductase, blocking the synthesis of DNA and arresting cells in the S phase. In sickle cell anemia, it promotes fetal hemoglobin (HbF) synthesis, improves red cell hydration, decreases neutrophil and platelet count, modifies red cell endothelial cell interactions and acts as a nitric oxide donor. Trials have shown the clinical benefit of hydroxycarbamide in a subpopulation of adult patients with SCD, with a 44% reduction in the median annual rate of painful crises, a decrease in the incidence of acute chest syndrome and an estimated 40% reduction in overall mortality over a 9-year observational period. Its use in pediatrics has also been well established; trials have shown it is well tolerated and does not impair growth or development. In addition it decreases the number and duration of hospital attendences. A number of emerging uses of hydroxycarbamide currently are being investigated, such as stroke prevention.