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National Institutes of Health Fact Sheet Sickle Cell Disease

Source: www.nih.gov
Topic: Sickle Cell Disease
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Sort Desciption: Thirty Years Ago As recently as 1970, the average patient with sickle cell disease (SCD) died in childhood, often of overwhelming infection. Approximately 10 percent of children with SCD suffered fatal or debilitating strokes. ...

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Thirty Years Ago As recently as 1970, the average patient with sickle cell disease (SCD) died in childhood, often of overwhelming infection. Approximately 10 percent of children with SCD suffered fatal or debilitating strokes. Although the technology for screening newborns for SCD was available, it was not generally used because an early diagnosis offered no advantage. Today Today, patients with sickle cell anemia live to their mid-40s and patients with a related condition, SC-hemoglobin disease, live to the mid-60s. This remarkable improvement can be attributed, in large part, to NIH research. When NIH-supported researchers discovered that a daily dose of penicillin could prevent fatal infections in infants who had SCD, they not only established a new standard of care but also provided an impetus for widespread neonatal screening. Today, newborns found to have the disease are given antibiotics until age 5, when prophylaxis can be stopped safely (as demonstrated by another NIH study). With NIH support, researchers found ways to identify children with SCD who were likely to have strokes and established that regular blood transfusions could reduce stroke risk by 90 percent. A subsequent study showed that, unlike the case with prophylactic penicillin, transfusion therapy must be continued indefinitely to maintain protection from stroke. Based on results of an NIH-supported clinical trial in adults, hydroxyurea became the first agent approved by the U.S. Food and Drug Administration (FDA) for prevention of painful sickle cell episodes. Hydroxyurea increases life expectancy, reduces emergency department visits and hospitalizations, and is cheaper than standard care for even the most ill of adult patients. Another study showed hydroxyurea to be safe and effective in children aged 5-15 years, and it is now being tested in even younger ...

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