Lessons from the stroke prevention trial in sickle cell anemia (STOP) study
Lessons from the stroke prevention trial in sickle cell anemia (STOP) study
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DOI：
10.1177/088307380001500511
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发表时间：
2000-05-01
影响因子：
1.9
通讯作者：
Adams, RJ
中科院分区：
文献类型：
--
作者：
Adams, RJ
中风在儿童中通常并不常见，但镰状细胞病与儿童早期中风的高风险相关。大脑动脉，特别是大脑中动脉和颅内颈内动脉，会发生狭窄，容易发生缺血性中风。使用经颅多普勒超声检查进行无创风险预测使得在比较长期输血与标准护理的临床试验中测试初级中风预防成为可能。由 14 个临床中心组成的联盟进行了一项随机临床 Mal（镰状细胞性贫血中风预防 -“STOP”研究），以测试预防镰状细胞病儿童首次中风的策略。超过 2000 名儿童接受了经颅多普勒超声检查，其中 130 名血流速度升高表明高风险的儿童被纳入试验。定期输注红细胞足以将 Hb S 基因产物占总血红蛋白的百分比从 90 以上降至 30 以下，与中风的显着减少相关。每年未经治疗的风险为 10%，经过治疗后风险降低了 90% 以上，这一效果足以导致试验提前终止。尽管治疗是非盲法，但设计包括由远离研究地点的神经科医生小组对可能的中风进行盲法裁决。该研究引发了国家心肺血液研究所发布的临床警报，建议对患有镰状细胞病的儿童以及根据经颅多普勒超声检查存在风险且未患过中风的 2 至 16 岁儿童进行筛查和考虑治疗。
Stroke is generally uncommon in children, but sickle cell disease is associated with a high risk of stroke in the early yeats of childhood. Large cerebral arteries, especially the middle cerebral and intracranial internal carotid, develop stenosis that predisposes to ischemic stroke. Noninvasive prediction of risk using transcranial Doppler ultrasonography made it possible to test primary stroke prevention in a clinical trial comparing chronic blood transfusion with standard care. A consortium of 14 clinical centers conducted a randomized clinical Mal (Stroke Prevention in Sickle Cell Anemia-the "STOP" study) to test a strategy to prevent first stroke in children with sickle cell disease. Over 2000 children were screened with transcranial Doppler ultrasonography and of these, 130 with elevated blood velocity indicating high risk were enrolled in the trial. Regular red cell transfusions sufficient to reduce the percentage of Hb S gene product from over 90 to less than 30 of total hemoglobin was associated with a marked reduction in stroke. The untreated risk of 10% per year was reduced over 90% with treatment, an effect sufficient to cause early termination of the trial. Although treatment was unblinded, the design included blinded adjudication of possible stroke by a panel of neurologists remote from the study sites. The study led to a Clinical Alert, issued by the National Heart, Lung, and Blood Institute, recommending screening and consideration of treatment in children with sickle cell disease and 2 to 16 years of age who are at risk based on transcranial Doppler ultrasonography, and who have not had stroke.