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Hydroxyurea therapy improves survival in most severely affected sickle cell patients

-approved drug for the treatment of sickle cell anemia.

In the new long-term "observational" study, patients selected with their physicians whether to continue, start, or stop treatment with hydroxyurea. Seventy-five (25 percent) of the original 299 patients died during the 9-year study. According to study authors, this statistic reflects the severity of the disease as required by the study design, and the high death rate in adults with sickle cell anemia.

Among study participants, the death rate was 28 percent for those with the lowest fetal hemoglobin level compared to a 15 percent death rate for those with the highest level of fetal hemoglobin. Fetal hemoglobin inside red blood cells of sickle cell anemia patients may prevent these cells from becoming sickled and rigid, hallmarks of the inherited disease. Hydroxyurea is believed to stimulate the production of fetal hemoglobin.

The new study also found that patients with 3 or more painful episodes per year during the trial had a 27 percent death rate versus a 17 percent death rate for those with less frequent episodes.

"Hydroxyurea's ability to reduce complications in the sickest patients is strongly connected to lower death rates," said Duane Bonds, M.D., study co-author and project officer for the MSH Patients' Follow-up Study. "These results also confirm that the benefits of hydroxyurea persist. After the original trial ended, we didn't know if the benefits of the drug would be long term," added Bonds. The new study also has important implications for significantly decreasing health care costs, said Bonds. Patients with more severe forms of the disease are seen in the emergency room and are hospitalized more frequently than patients who are less severely affected.

According to lead investigator Dr. Martin Steinberg, who is Director of the Center of Excellence in Sickle Cell Disease at Boston University School of Medicine, the latest research has important implications for
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Contact: NHLBI Communications Office
301-496-4236
NIH/National Heart, Lung, and Blood Institute
1-Apr-2003


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