Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia

Article Abstract:

The drug hydroxyurea may be useful in preventing sickle cell crises. Sickle cell crisis occurs when sickled red blood cells block blood vessels, causing pain in the affected area. Of 299 men and women with sickle cell anemia, 152 began taking 15 milligrams per kilogram of hydroxyurea each day and 147 took a placebo. The dose of hydroxyurea was gradually increased to determine the maximally tolerated dose. Although the trial was planned to last two years, it was stopped when the benefits of hydroxyurea treatment became clear four months after treatment. Hydroxyurea treatment reduced the median annual rate of sickle cell crises by 44%, from 4.5 crises a year in those taking placebo to 2.5 crises a year in those taking hydroxyurea. Patients taking hydroxyurea also had fewer other complications of sickle cell anemia and received fewer blood transfusions. However, almost all experienced bone marrow suppression and had to stop taking the drug temporarily. Lower doses may be just as effective and cause fewer side effects.

Evaluation, Hydroxyurea

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Reversing ontogeny

Article Abstract:

Increasing fetal hemoglobin production in patients with beta-thalassemia and sickle cell disease may be an effective form of treatment. Normally, production of fetal hemoglobin nearly stops before birth, and adult hemoglobin production dominates. While increased fetal hemoglobin production does not address the underlying genetic mutations behind beta-thalassemia and sickle cell anemia, it does compensate for the hemoglobin disorders and eases the symptoms. Most efforts to stimulate fetal hemoglobin have centered on hydroxyurea, but its effects are limited and its long-term use may induce tumor formation. A recent study found that erythropoietin taken in combination with hydroxyurea improved fetal hemoglobin production. Preliminary studies on butyrate suggest it may be even safer and more effective than hydroxyurea in elevating fetal hemoglobin. The prospect of gene therapy for these hemoglobin disorders remains distant, but improvements in drug treatment are promising.

Editorial, Thalassemia

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