Prevention of conversion to abnormal transcranial Doppler with hydroxyurea in sickle cell anemia: A Phase III international randomized clinical trial

Prevention of conversion to abnormal transcranial Doppler with hydroxyurea in sickle cell anemia: A Phase III international randomized clinical trial

Hankins JS (2015), American Journal of Hematology (Link to Abstract)                                                     


Cerebrovascular stroke is a known complication of sickle cell disease. Previous studies have established monitoring transcranial doppler ultrasound velocities as an effective way to monitor the risk of stroke in these patients. Currently, treatment is initiated for those children at highest risk of stroke (TCD velocity >200 cm/sec), while those with conditional velocities (170-199 cm/sec) are observed. This is a prospective randomized control trial comparing TCD ultrasound velocities in sickle cell patients with conditional TCD velocities who were and were not treated with hydroxyurea.



38 children were enrolled on the trial. There was a significant difference in conversion of conditional TCD velocities to abnormal or high risk velocities (>200 cm/sec) between the treatment and control arms by post hoc analysis (9% in the treatment group compared and 47% in the control group, respectively, P=0.02), though no strokes occurred in either group. In addition, the treatment group showed a significant decrease in their mean TCD velocities (P=0.02). Unfortunately, the study had to be closed early due to difficulties with patient accrual, and therefore the numbers were insufficient to prove significant prevention of progression by intention-to-treat analysis.