Credit: St Jude Children’s
Research Hospital
Monthly blood transfusions can reduce the risk of silent or overt stroke among children with sickle cell disease (SCD) who previously had a silent stroke, according to a study published in The New England Journal of Medicine.
Children with evidence of silent cerebral infarcts who received monthly blood transfusions for 3 years had a 58% lower risk of suffering repeat silent or overt
strokes than children who did not receive transfusions.
In fact, researchers said the actual benefit of transfusion therapy may be even higher, as 15% of the children who were assigned to receive transfusions either did not receive them or only received them for a brief period.
“The results of our study show that blood transfusions can play a critical role in preventing this insidious and potentially devastating condition,” said study author James F. Casella, MD, of the Johns Hopkins Children’s Center in Baltimore, Maryland.
“They also highlight the importance of intervening early to preclude ongoing or further brain injury among these youngsters. Most importantly, our findings suggest a much-needed treatment option for clinicians and families of children with sickle cell disease who have had silent strokes.”
Previous studies have suggested that blood transfusions may help prevent stroke in patients with SCD by increasing the number of normal red blood cells and decreasing the likelihood of blocked blood vessels.
But Dr Casella and his colleagues wanted to determine if monthly blood transfusions would help prevent stroke in children with SCD who had evidence of a previous silent cerebral infarct, as well as whether the benefits of transfusion outweigh the risks.
The researchers analyzed 196 children, ages 5 to 15 years, who were diagnosed with SCD and had infarct-like lesions on their MRI scans. The children were randomized to an observation arm or to receive blood transfusions every month for 3 years.
Six percent (6/99) of children who received regular transfusions suffered another silent or overt stroke. One of the patients had a stroke, and 5 had new or enlarged silent cerebral infarcts.
In comparison, 14% (14/97) of children in the observation arm experienced a silent or overt stroke. Seven had a stroke, and 7 had new or enlarged silent cerebral infarcts.
So children who did not receive transfusions were more than twice as likely as their peers to have repeat strokes.
Children who did not receive transfusions were also more likely to suffer a range of other SCD-related problems, such as episodes of extreme pain. There were 295 pain episodes among children who did not receive transfusions and 126 episodes among transfused patients.
An unexpected result, according to the researchers, was that intelligence measures were not different between the 2 treatment arms. Previous studies suggested that silent strokes are associated with a 5-point reduction in IQ. The researchers said they plan to explore this finding further.
Nevertheless, this study provides “clear evidence” that transfusions can decrease the progression of silent strokes in children with SCD, said study author Michael R. DeBaun, MD, of Vanderbilt University in Nashville, Tennessee.
“These results suggest that children who have this disease should be screened early for silent strokes, at least by the time they begin elementary school, to help them manage the disease and to ensure minimal impact on school performance,” he added.
Dr DeBaun and his colleagues said children with SCD should have a surveillance MRI, preferably without sedation, at a young age. Most children with SCD who are at risk for a silent stroke will have one by age 6 years.
The researchers also noted that healthcare providers should discuss treatment options with families to determine if transfusion therapy is appropriate, as there is a risk of transfusion reactions and iron overload.
The decision to transfuse should be made by factoring in each child’s overall health, medical history, and the ability to take time from school for monthly procedures.
The researchers said further study is needed to identify which children with a history of silent strokes are at greatest risk for recurrence so transfusion therapy can be targeted to them.
An editorial related to this study also calls for additional research to determine if the findings can be translated to clinical practice.
