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Regular transfusions reduced stroke recurrence in pediatric sickle cell anemia
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Children with sickle cell anemia who underwent regular blood-transfusion therapy experienced fewer recurrences of cerebral infarct, according to results of a single-blind study.
“The results of our study show that blood transfusions can play a critical role in preventing this insidious and potentially devastating condition,” James F. Casella, MD, Rainey professor of pediatric hematology at Johns Hopkins Children’s Center, said in a press release. “Our findings suggest a much-needed treatment option for clinicians and families of children with sickle cell disease who have had silent strokes.”
James F. Casella
Casella and colleagues evaluated data from 196 children aged 5 to 15 years who had at least one infarct-like lesion on an MRI scan. Researchers randomly assigned 99 patients to receive regular transfusions, and the other 97 patients underwent observation.
The recurrence of a stroke or a new or enlarged silent cerebral infarct served as the study’s primary endpoint.
Median follow-up was 3 years. During that time, 90 patients assigned the intervention underwent transfusions, and 95% of these patients repeated the procedure within 38 days.
Six patients (6%) assigned transfusions experienced an event. One patient had a stroke, and five had a new or enlarged silent cerebral infarct.
In the observation group, 14 patients (14%) experienced an event. Seven had strokes and seven had new or enlarged silent cerebral infarcts.
The incidence of the primary endpoint in the intervention arm was two events per 100 years at risk, which was significantly lower than the incidence of 4.8 events per 100 years at risk in the observation arm (incidence risk ratio [IRR]=0.41; 95% CI, 0.12-0.99).
Researchers calculated a relative risk reduction of 58% with the regular transfusions. Thirteen patients needed to undergo treatment for 3 years to prevent one infarct recurrence.
Infarct recurrence was more common among patients who were younger (OR=1.41; 95% CI, 1.12-1.78), those who had a history of recurring headaches (OR=4.33; 95% CI, 1.50-13.06) and those who had a higher steady-state reticulocyte count (OR=1.11; 95% CI, 1.01-1.22) at baseline.
Significantly more patients in the observation arm experienced vaso-occlusive pain (IRR=0.41; 95% CI, 0.20-0.75), acute chest syndrome (IRR=0.13; 95% CI, 0.04-0.28), priapism (IRR=0.13; 95% CI, 0.03-0.55) and symptomatic avascular necrosis of the hip (IRR=0.22; 95% CI, 0.05-0.85) than those who underwent transfusion.
Seventeen percent of patients who underwent transfusion experienced a reaction, and more than half of those (52%) were allergic reactions.
“The study indicates that screening for silent strokes in children starting school can lead to early detection and prevention of recurrences, as well as reduction in other complications of sickle cell anemia such as acute painful episodes and acute chest syndrome,” Deborah Hirtz, MD, program director of the National Institute of Neurological Disorders and Stroke of the NIH, said in a press release. “The results of this trial will make a difference to children with sickle cell anemia and their families.”
Disclosure: The researchers report honoraria, grant support, travel support, lecture fees, and data and safety monitoring board fees from; advisory board and consultant roles with; and pending royalties on patents licensed to Eli Lilly, Guerbet, ImmunArray, Mast Therapeutics, Novartis, Resonance Health, Shire Pharmaceuticals, Sideris Pharmaceuticals and Siemens Healthcare.
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