Harvey Luksenburg, Ph.D. National Heart, Lung, & Blood Institute two years of the initial event. The NIH’s Role in the Prevention and Reduction of Strokes in Children with Sickle Cell Disease ( )

Stroke in Children with Sickle Cell Disease  Stroke is a leading cause of death and disability in both children and adults with SCD.  The risk of having a stroke up until the age 20 years is 11%.  This risk is about 300 times higher than seen in children without SCD.  Recurrent stroke occurs in about two-thirds of children within two years of the initial event.

 TCD is a non-invasive test that measures the velocity of blood flow in large vessels of the brain.  TCD is used to identify children with a high risk of stroke. Transcranial Doppler Ultrasonography (TCD)

 Children with a high risk for stroke (TCD) were randomized to receive either regular blood transfusions or standard supportive care.  After one year 10 children in the standard care group had a stroke, but only one child in the transfusion group had a stroke.  This represented a 90% decline in the stroke rate. The Stroke Prevention Trial in Sickle Cell Disease (STOP) ( )

 As a result of these findings, NIH recommended that the study be terminated one year early.  Recommendations: children with SCD who are identified at high risk for stroke should receive regular red cell transfusions. STOP Trial

 STOP 2 Trial (2005): If TCD normalizes can transfusions be discontinued?  Children receiving transfusions for stroke prevention for at least 30 months.  Randomized to either continued transfusion or no transfusion. How Long Should Transfusions Be Given?

 In the group that had transfusion stopped, 14 developed a high TCD (high risk for stroke) and 2 had strokes.  In the group that continued transfusion, no child developed either a stroke or a high TCD.  Conclusion: Transfusion should be continued indefinitely in children at high risk for stroke. STOP 2--Results

 Children’s Hosp. of Philadelphia reported a 90% reduction in acute strokes in children through a program of TCD screening and transfusion.  This resulted in an increase in the number of children receiving regular blood transfusions. J.Pediatrics 2010 Reduction in Stroke in Children

 Can strokes in children be prevented by hydroxyurea (instead of transfusions)?  What are the optimal ways to predict and treat stroke in adults? Questions for Further Research;