A medication traditionally associated with the treatment of cancer and AIDS has emerged as a surprise weapon in the fight against sickle-cell anemia, the painful and often fatal inherited blood disorder that occurs primarily in African Americans. A new research study by the National Heart, Lung, and Blood Institute (NHLBI) has found that the drug hydroxyurea can significantly improve the survival rate for sickle-cell patients suffering from the most severe forms of the disease. These patients typically die 10 to 15 years earlier than those with milder cases. Although previous research had shown hydroxyurea to be effective in reducing the symptoms of severely ill sickle-cell patients, this was the first study to evaluate whether the treatment prolongs life.


The long-term “observational” study followed 299 adult sickle-cell patients with moderate to severe cases of the disease over a nine-year period. The findings, published in the April 2 issue of The Journal of the American Medical Association, showed that the patients who used hydroxyurea were 40% less likely to die during the study than those who did not use the drug.

The lower death rates were found to be strongly connected to hydroxyurea’s ability to reduce sickle-cell symptoms and complications. Specifically, the drug helps reduce episodes of severe pain crises and chest syndrome, a pneumonia-like illness. Hydroxyurea is also believed to stimulate the production of fetal hemoglobin, which may help prevent red blood cells from becoming sickle-shaped and rigid.

Currently, many patients who could benefit from hydroxyurea are not being treated with the drug, reports the study’s lead investigator, Dr. Martin Steinberg, director of the Center of Excellence in Sickle-Cell Disease at Boston University School of Medicine. “Our study strongly suggests that adults with sickle-cell anemia and clinically significant complications of their disease should take hydroxyurea under the supervision of a knowledgeable physician,” he says. “Most people who take this medication will feel better; we now know they will also live longer.”

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