Short‐course blood transfusion therapy with hydroxyurea, a functional strategy in the management of stroke in children with sickle cell disease

Summary Cerebrovascular accidents are serious complications of sickle cell disease (SCD). Children with abnormal transcranial Doppler (TCD) readings are at higher risk for stroke, and those with prior strokes have increased risk of recurrence. Chronic blood transfusion therapy (BTT) is the standard treatment for stroke prevention; hydroxyurea (hydroxycarbamide [HU]), a recommended alternative, is less effective. This study explores the use of modified short BTT combined with HU for stroke prevention in SCD. We reviewed medical records of 170 children (ages 2–16) with abnormal TCD or prior stroke treated with monthly BTT for 6–8 months and HU. TCD results and stroke incidence were assessed 1 year after discontinuing BTT. Among the children, 136 (80%) had abnormal TCD, 15 (11.2%) had current stroke and 19 (8.8%) had prior stroke. After discontinuation of BTT, abnormal TCD readings decreased significantly to 18.7% and 16.9% ( p  < 0.05); those with normal TCD increased significantly from 38.8% to 52.8% ( p  < 0.05) after 1 year. TCD velocity decreased significantly during the same period ( p  < 0.05). Only 2 (1.5%) children with abnormal TCD experienced stroke. Overall, 13 (7.8%) had stroke by 1‐year post BTT. In conclusion, the combination of short‐course BTT and HU significantly reduced stroke risk and incidence of stroke in children with SCD.