Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease

BACKGROUND: Prophylactic transfusion prevents strokes in children with sickle cell anemia who have abnormalities on transcranial Doppler ultrasonographic examination. However, it is not known how long transfusion should be continued in these children. METHODS: We studied children with sickle cell disease who had a high risk of stroke on the basis of a transcranial Doppler screening examination and who had received transfusions for 30 months or longer, during which time the Doppler readings became normal. The children were randomly assigned to continued transfusion or no continued transfusion. Children with severe stenotic lesions on cerebral magnetic resonance angiography were excluded. The composite primary end point was stroke or reversion to a result on Doppler examination indicative of a high risk of stroke. RESULTS: The study was stopped after 79 children of a planned enrollment of 100 underwent randomization. Among the 41 children in the transfusion-halted group, high-risk Doppler results developed in 14 and stroke in 2 others within a mean (+/-SD) of 4.5+/-2.6 months (range, 2.1 to 10.1) of the last transfusion. Neither of these events of the composite end point occurred in the 38 children who continued to receive transfusions. The average of the last two transcranial Doppler results before transfusion was started was the only predictor of the composite end point (P=0.05). CONCLUSIONS: Discontinuation of transfusion for the prevention of stroke in children with sickle cell disease results in a high rate of reversion to abnormal blood-flow velocities on Doppler studies and stroke. (ClinicalTrials.gov number, NCT00006182.)