Improved treatment for Sickle Cell

Improved treatment for Sickle Cell

(WCSC) - Some children with sickle cell disease may be able to avoid blood transfusions and take medication instead.

A national study involving the Medical University of South Carolina found the drug hydroxyurea as effective as blood transfusions to reduce blood flow speeds in the brain.

The study found the therapy so effective, the research study was stopped early because of the positive results.

Increased blood flows are a major risk factor for stroke in children with sickle cell. Standard treatment for children with sickle cell disease who are at high risk of stroke consists of regular blood transfusions.

Children who receive regular blood transfusions are then at risk for iron overload. Iron-reduction therapy is needed for those receiving transfusions.    

The National Institutes of Health (NIH)-supported study sought to answer whether hydroxyurea would provide the same benefit as blood transfusions.

Researchers from 26 clinical studied 121 children ages 4 to 16 years old and divided them into two groups, one that received transfusions and one that was transitioned from transfusions to daily doses of hydroxyurea.

"This study shows that some children can be moved from transfusion to medication after at least a year," said Robert Adams, M.D. study principal investigator.

Researchers are working toward the National Institutes' goal of a 'stroke free generation' in sickle cell disease.  The findings were published in the journal, The Lancet, and presented at the American Society of Hematology.

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