Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia.

N Engl J Med 2014;371:699-710.
N Engl J Med 2014;371:699-710.
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Review by : L. T. Goodnough
NATA Review

This randomised, controlled study of children (5-15 years of age) with sickle cell anaemia studied the value of chronic RBC transfusion support (to maintain a target haemoglobin concentration greater than 9 g/dL and a target haemoglobin S less than 30% of total haemoglobin) compared with standard of care (observation group).

The primary endpoint, recurrence of infarct or haemorrhage as determined by neuroimaging, was reached in 6% and 14% of the patients, respectively, with a calculated incidence of 2.0 and 4.8 events per 100 years of risk (P = 0.04).

The implications of this study of transfusion therapy and long-term complications such as iron overload, when compared to other pharmalogic options such as hydroxyurea therapy, are discussed in an accompanying editorial.

– Lawrence Tim Goodnough

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