PUBLIC RELEASE DATE:

20-Aug-2014

Contact: Ashley Culver ashley.culver@vanderbilt.edu 615-322-4747 Vanderbilt University Medical Center

Vanderbilt-led research, as part of an international, multicenter trial, found regular blood transfusion therapy significantly reduces the recurrence of silent strokes and strokes in children with sickle cell anemia who have had pre-existing silent strokes, according to study results released today in the New England Journal of Medicine.

Michael R. DeBaun, M.D., MPH, director of the Vanderbilt-Meharry Sickle Cell Disease Center of Excellence and professor of Pediatrics, was the principal investigator of the more than $20 million, federally-funded trial, the largest of its kind in children with sickle cell. Nearly 100,000 people in the United States are living with sickle cell disease, a group of inherited blood disorders.

Sickle cell disease most commonly affects African-Americans, occurring in one of every 396 births in this ethnic group. In patients with the disease, red blood cells are abnormal hard, sticky and shaped like a crescent moon. When the sickle cells travel through small blood vessels, they can decrease the normal blood flow to all organs of the body, causing many complications including pain that require hospitalization for treatment, lung disease and a shortened life span.

Silent strokes are frequent in sickle cell anemia (the most common form of sickle cell disease), occurring in approximately 3 percent of school-age children with the disease, and can cause poor school performance and limit performance of complex tasks. The only way to diagnose a silent stroke is with magnetic resonance imaging (MRI) of the brain. Once a child has had a silent stroke, he is at a much higher risk for more dangerous overt strokes and new or enlarged silent stokes.

Results of the 10-year trial led by DeBaun, which involved 29 clinical centers in the United States, France, Canada and the United Kingdom, appear in the Aug. 21 issue of NEJM.

The Silent Cerebral Infarct Transfusion (SIT) Trial looked at the efficacy of blood transfusion therapy for children with sickle cell anemia to prevent repeat cerebral infarcts (overt strokes and silent strokes). Researchers found that monthly blood transfusions reduced the recurrence of silent strokes and strokes by 58 percent in children with pre-existing silent strokes when compared to children who were not transfused. The actual benefit of transfusion therapy is probably higher because 15 percent of the children who were assigned to receive blood transfusion therapy either never received the transfusion or only received transfusions for a brief period, but were counted as if they were transfused, referred to as "intention to treat analysis."

"The success of the trial is a tribute to over 1,000 families of children who were screened with an MRI of the brain and the almost 200 families of children who committed to monthly visits for three years. The successful recruitment, adherence and retention of black families, many of whom were working poor, to an arduous, complex trial, should lay to rest the misperception that blacks are not willing participants in research," said DeBaun, J.C. Peterson M.D. Professor of Pediatric Pulmonology. "The results of the trail indicate members of the community are interested in research, when they are well-informed, empowered to make their own decision, and have a trusting relationship with the health care provider who is independent of their participation in the study," DeBaun continued.

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Regular blood transfusions can reduce repeat strokes in children with sickle cell disease

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