Abstract：
Sickle cell disease (SCD) increases the incidence of childhood stroke eighty-fold. Stroke risk can be estimated by measurement of the blood velocity through the middle cerebral artery (MCA) using transcranial doppler ultrasound (TCD). A high MCA blood velocity indicates increased stroke risk due to cerebral vasculopathy, and first-line treatment to prevent primary or recurrent strokes in high-risk children with SCD has classically been chronic blood transfusions. Research has more recently shown that many of these patients may safely transition from transfusions to oral hydroxyurea (HU) treatment while maintaining a decreased risk of stroke. However, the effect on stroke risk of truly prophylactic HU treatment beginning in infancy, prior to the onset of cerebral vasculopathy, is less well understood. Our retrospective study aimed to document the long-term effects of HU treatment compared with no HU treatment in children with SCD, using TCD measurements as our primary outcome and a surrogate marker of stroke risk. Our results showed that when accounting for age-related variability and duration of treatment, prophylactic HU treatment was independently associated with lower TCD MCA velocities compared with no HU treatment, providing further evidence supporting its early initiation for patients with SCD.
摘要：
镰状细胞病（SCD）使儿童中风的发病率增加了八十倍。可以通过使用经颅多普勒超声（TCD）测量通过大脑中动脉（MCA）的血流速度来估计中风风险。较高的MCA血流速度表明脑血管病变导致的卒中风险增加，在高危SCD儿童中预防原发性或复发性卒中的一线治疗通常是慢性输血。最近的研究表明，这些患者中的许多人可以安全地从输血过渡到口服羟基脲（HU）治疗，同时保持中风风险的降低。然而,从婴儿期开始的真正预防性HU治疗对中风风险的影响，在脑血管病变发作之前，不太了解。我们的回顾性研究旨在记录在SCD患儿中使用HU治疗与不使用HU治疗相比的长期效果。使用TCD测量作为我们的主要结局和卒中风险的替代指标。我们的结果表明，当考虑到年龄相关的变异性和治疗持续时间时，与没有HU治疗相比，预防性HU治疗与较低的TCDMCA速度独立相关，提供进一步的证据支持早期开始治疗SCD患者。
