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Hydroxyurea Endorsed for Sickle Cell Disease

Consensus statement cites strong evidence supporting its use in adults, acceptable risk profile

THURSDAY, May 8 (HealthDay News) -- In the treatment of sickle cell disease, hydroxyurea represents a major advance, according to a National Institutes of Health Consensus Development Conference Statement published online May 6 in the Annals of Internal Medicine.

Otis W. Brawley, M.D., of Emory University in Atlanta, and other members of an independent panel based their statement on a systematic literature review conducted under contract with the Agency for Healthcare Research and Quality, and presentations by clinical investigators at the National Institutes of Health Consensus Development Conference held Feb. 25-27, 2008 in Bethesda, Md.

The investigators found that hydroxyurea is backed by strong evidence supporting its use in adults to decrease severe pain episodes, hospitalizations, number of blood transfusions and acute chest syndrome. Although they found weaker evidence to support its use in children, they said that preliminary data are encouraging. They also found reassuring evidence about the short-term and long-term risks of hydroxyurea therapy and called them "acceptable" compared to the risks of untreated sickle cell disease.

"It is difficult to draw conclusions about the effectiveness of hydroxyurea in everyday practice because we lack precise estimates of the number of people with sickle cell disease in the United States and the number of people receiving hydroxyurea," the authors write. "Furthermore, although barriers to the use of hydroxyurea in persons with sickle cell disease seem to be extensive, little research exists on these patient-, parent/family/caregiver-, provider-, and system-level barriers. More studies are required to address these issues."

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