Background Prophylactic transfusion prevents strokes in childrenwith sickle cell anemia who have abnormalities on transcranialDoppler ultrasonographic examination. However, it is not knownhow long transfusion should be continued in these children.
Methods We studied children with sickle cell disease who hada high risk of stroke on the basis of a transcranial Dopplerscreening examination and who had received transfusions for30 months or longer, during which time the Doppler readingsbecame normal. The children were randomly assigned to continuedtransfusion or no continued transfusion. Children with severestenotic lesions on cerebral magnetic resonance angiographywere excluded. The composite primary end point was stroke orreversion to a result on Doppler examination indicative of ahigh risk of stroke.
Results The study was stopped after 79 children of a plannedenrollment of 100 underwent randomization. Among the 41 childrenin the transfusion-halted group, high-risk Doppler results developedin 14 and stroke in 2 others within a mean (±SD) of 4.5±2.6months (range, 2.1 to 10.1) of the last transfusion. Neitherof these events of the composite end point occurred in the 38children who continued to receive transfusions. The averageof the last two transcranial Doppler results before transfusionwas started was the only predictor of the composite end point(P=0.05).
Conclusions Discontinuation of transfusion for the preventionof stroke in children with sickle cell disease results in ahigh rate of reversion to abnormal blood-flow velocities onDoppler studies and stroke. (ClinicalTrials.gov number, NCT00006182
[ClinicalTrials.gov]
.)
Source Information
Robert J. Adams, M.D., and Donald Brambilla, Ph.D., of the STOP 2 investigative team take responsibility for the content of this article.
Address reprint requests to Dr. Adams at the Department of Neurology, Medical College of Georgia, 1429 Harper St., HF 1154, Augusta, GA 30912, or at rjadams{at}mcg.edu.
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