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Hydroxyurea therapy lowers transcranial Doppler flow velocities in children with sickle cell anemia Zimmerman SA; Schultz WH; Burgett S; Mortier NA; Ware REBlood 2007[Aug]; 110 (3): 1043-7Hydroxyurea has hematologic and clinical efficacy in sickle cell anemia (SCA), but its effects on transcranial Doppler (TCD) flow velocities remain undefined. Fifty-nine children initiating hydroxyurea therapy for clinical severity had pretreatment baseline TCD measurements; 37 with increased flow velocities (> or = 140 cm/s) were then enrolled in an institutional review board (IRB)-approved prospective phase 2 trial with TCD velocities measured at maximum tolerated dose (MTD) and one year later. At hydroxyurea MTD (mean +/- 1 SD = 27.9 +/- 2.7 mg/kg per day), significant decreases were observed in the right middle cerebral artery (MCA) (166 +/- 27 cm/s to 135 +/- 27 cm/s, P < .001) and left (MCA) (168 +/- 26 cm/s to 142 +/- 27 cm/s, P < .001) velocities. The magnitude of TCD velocity decline was significantly correlated with the maximal baseline TCD value. At hydroxyurea MTD, 14 of 15 children with conditional baseline TCD values improved, while 5 of 6 with abnormal TCD velocities whose families refused transfusions became less than 200 cm/s. TCD changes were sustained at follow-up. These prospective data indicate that hydroxyurea can significantly decrease elevated TCD flow velocities, often into the normal range. A multicenter trial is warranted to determine the efficacy of hydroxyurea for the management of increased TCD values, and ultimately for primary stroke prevention in children with SCA.|*Ultrasonography, Doppler, Transcranial[MESH]|Anemia, Sickle Cell/complications/diagnostic imaging/*physiopathology/therapy[MESH]|Antisickling Agents/*administration & dosage[MESH]|Blood Flow Velocity/*drug effects[MESH]|Blood Transfusion[MESH]|Child[MESH]|Child, Preschool[MESH]|Female[MESH]|Humans[MESH]|Hydroxyurea/*administration & dosage[MESH]|Male[MESH]|Maximum Tolerated Dose[MESH]|Middle Cerebral Artery/diagnostic imaging[MESH]|Prospective Studies[MESH]|Stroke/diagnostic imaging/etiology/*physiopathology/prevention & control[MESH] |
