Sickle cell disease, a congenital inherited disorder, has severe consequences and usually shortens life while causing significant pain and disability. Around 0.3 percent of black Americans have sickle disease, which is inherited by 25 percent of children whose parents both carry the gene. Between 8 and 13 percent carry one copy of the gene, the Merck Manual reports. While no cure for sickle cell disease exists, treatment with hydroxyurea helps reduce symptoms and improve complications. Hydroxyurea does have side effects and restrictions on use due to potential damage.
Action
Hydroxyurea increases the body's production of fetal hemoglobin, which blocks the sickling action of cells in sickle cell anemia. However, hydroxyurea, a chemotherapy agent, also suppresses white blood cell and platelet production, the University of Maryland Medical Center (UMMC) explains.
Population
Hydroxyurea is prescribed for use mainly in adults and adolescents, although studies show the drug to be relatively safe for use even in infants, the UMMC states. Hydroxyurea is a category D drug in pregnancy and, therefore, should not be taken by women who are pregnant or who might become pregnant. Breastfeeding mothers also should not take the drug, the University of California San Diego warns.
Benefits
Hydroxyurea reduces the incidence of painful vaso-occlusive crises caused by blockage of blood vessels with sickled cells by 50 percent, according to the Merck Manual. In a study carried out by Harvard University Brigham and Women's Hospital in 1998, hydroxyurea also reduced the chance of developing acute chest syndrome in half. Effectiveness of the drug appears to continue indefinitely as long as it is taken.
Side Effects
Because hydroxyurea is a chemotherapy agent, it has many potential side effects. Typical effects include hair loss, nausea, constipation, drowsiness and mouth inflammation. Low platelet count, leading to possible bleeding episodes and low white blood count---which increases susceptibility to infection---can also occur. Long-term use may increase the risk of developing leukemia, a cancer of white blood cells, the UMMC warns. Skin rashes, headaches and diarrhea may also occur.
Considerations
While hydroxyurea can reduce painful sickling episodes that lead to frequent hospitalizations, the drug can also cause harmful and potentially life-threatening side effects. Weighting the risks and benefits before beginning treatment is essential. Safety for use in children has not been well established; the drug may interfere with growth. The drug must be handled with care and should not be handled by people other than those taking the drug. Getting the drug on the skin, eyes, nose or mouth can be dangerous, the University of California San Diego warns, so wear gloves when handling the medication.
