Bristol-Myers Squibb's trial of Hydrea (hydroxyurea) in sickle cell anemia has been halted early by the National Heart, Lung and Blood Institute in the USA because the results are so compelling. The NHLBI has sent out a clinical alert to US physicians informing them of the results, which are believed to be the first evidence of an effective treatment for the disorder.
Hydroxyurea treatment halved the number of painful sickle cell episodes, the frequency of acute chest syndrome and the number of disease-associated blood transfusions. The trial included a total of 299 adult sickle cell sufferers, who had experienced at least three painful sickle episodes in the last 12 months. Each patient was started at a dose of 15mg/kg, which was increased in increments of 5mg/kg every 12 weeks until toxicity or the maximum allowable dose of 35mg/kg was reached.
Reversible bone marrow suppression was the only significant toxicity observed, although the clinical alert cautions that hydroxyurea may not be appropriate therapy for all patients, as it is a cytotoxic agent and could be dangerous, eg in women likely to become pregnant or in those unwilling or unable to follow direction regarding treatment. There have been some suggestions that protracted hydroxyurea may be associated with an increased risk of leukemia, so follow-up monitoring of the sickle cell patients is continuing.
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