The study of 299 adult sickle cell patients with moderate to severe forms of the disease, found that improved survival was related to the benefits associated with hydroxyurea treatment an increase in fetal hemoglobin and reduced episodes of severe pain "crises" and chest syndrome, a pneumonia-like illness. The results were published in the April 2 issue of The Journal of the American Medical Association.
Previous research has shown the benefits of hydroxyurea in reducing the symptoms of the most severely affected sickle cell patients those who have 3 or more painful crises each year.
"The current study shows that hydroxyurea can improve the survival of the sickest patients. Now there is new hope for these patients, who typically die 10 to 15 years earlier than patients with milder cases," said NHLBI Director Claude Lenfant, M.D.
Patients in the new study were originally participants in the NHLBI's Multicenter Study of Hydroxyurea (MSH) in Sickle Cell Anemia. That clinical trial was designed to test whether hydroxyurea reduced symptoms of the disease compared to placebo in patients who had 3 or more crises per year. Patients were randomly assigned to hydroxyurea or placebo and the study was "double-blinded" indicating neither patients nor physicians knew who was taking the drug.
Over 2 1/ 2 years, the drug resulted in an almost 50 percent reduction in the number of painful crises and episodes of chest syndrome. Patients on hydroxyurea also required about 50 percent fewer transfusions and hospitalizations. These results, reported in 1995, were reviewed by the Food and Drug Administration (FDA) and hydroxyurea became the first FDA
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Contact: NHLBI Communications Office
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NIH/National Heart, Lung, and Blood Institute
1-Apr-2003