Ongoing transfusions needed to avoid strokes in children with sickle cell disease

The 10 percent of children with sickle cell disease who are at risk for a stroke need ongoing blood transfusions to reduce their risk, according to a study at 25 sites in North America.

The National Heart, Lung and Blood Institute of the National Institutes of Health, which funded the $11 million study headquartered at the Medical College of Georgia in Augusta, issued a clinical alert to coincide with the Dec. 5 announcement of study findings at the American Society of Hematology meeting in San Diego.

"Whatever process puts these children at risk is fairly durable," says Dr. Robert J. Adams, neurologist and stroke specialist at MCG and principal investigator on the Optimizing Primary Stroke Prevention in Children with Sickle Cell Anemia, or STOP II, study. "We believed that we had identified a group that might tolerate coming off transfusion but the results did not confirm this. Too many of those taken off had return of abnormal transcranial Doppler, the best indicator we have of stroke risk, and there were two strokes in this group. We need more research to come up with a better way to limit the use of transfusion while still preventing strokes."

"This important study shows the value of continuing periodic blood transfusions in preventing the serious and debilitating consequences of stroke," says NHLBI Acting Director Barbara Alving. "At the same time, there are risks of chronic transfusions and the decision to continue with this treatment must be made on a case-by-case basis."

The STOP II study tested whether regular transfusions could be safely stopped after at least 30 months in a group of children who reverted to low stroke risk based on normal transcranial Doppler and magnetic resonance angiography after treatment.

The study was to have enrolled 100 children. However, the NHLBI-appointed Data and Safety Monitoring Board recommended early closure of the study after 79 children were enrolled and randomized half cont

Contact: Toni Baker
Medical College of Georgia

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