Stroke prevention trial in sickle cell anemia
Stroke prevention trial in sickle cell anemia
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DOI：
10.1016/s0197-2456(97)00099-8
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发表时间：
1998-02-01
期刊：
影响因子：
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通讯作者：
Waclawiw, MA
中科院分区：
文献类型：
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作者：
Adams, RJ;McKie, VC;Waclawiw, MA
7-8% 患有镰状细胞病 (Hb SS) 的儿童会发生中风，是发病的主要原因。通过长期输血，复发率已从 46-90% 降至 10% 以下。然而，预防第一次中风会更好，因为即使是一次中风也可能导致不可逆转的脑损伤。经颅多普勒 (TCD) 超声可以检测与后续中风风险相关的动脉血流速率。通过结合 TCD 筛查和潜在有效的治疗，可以预防首次中风。镰状细胞性贫血中风预防试验 (STOP) 是第一个针对 Hb SS 的中风预防试验，也是第一个针对 Hb SS 进行随机、对照输血的试验。这项多中心试验旨在测试与标准治疗相比，通过定期输血将镰状血红蛋白降低至 30% 或更低是否能够将首次中风减少至少 70%。主要终点将是临床上明显的脑梗塞症状，与磁共振成像和血管造影（MRI/MRA）或症状性颅内出血的一致结果。次要终点是通过 MRI 在不涉及主要终点的大脑区域检测到的无症状脑损伤。该设计要求 6 个月的启动间隔、18 个月的 TCD 筛查和随机化，以及从入组到第 54 个月的卒中观察。该试验的主要特点是标准化 TCD 和 MRI/MRA 方案的盲解释以及终点的盲判定。样本量（每个治疗组 60 个）基于 TCD 速度与中风风险相关的前瞻性数据。大于或等于 200 厘米/秒的时间平均速度与 39 个月内 46% 的脑梗塞风险相关。样本量足以在 90% 功效下检测到主要终点减少 70%。该试验将确定输血对于中风的一级预防是否有效。次要目标可能会进一步了解输血对大脑的影响，并指导未来对 Hb SS 脑血管疾病的研究。 (C) 爱思唯尔科学公司 1998。
Stroke occurs in 7-8% of children with Sickle Cell Disease (Hb SS) and is a major cause of morbidity. Rates of recurrence have been reduced from 46-90% to less than 10% through chronic blood transfusions. Prevention of first stroke, however, would be preferable because even one stroke can cause irreversible brain injury. Transcranial Doppler (TCD) ultrasound can detect arterial blood flow rates associated with subsequent stroke risk. By combining TCD screening and a potentially effective treatment, first stroke may be prevented. The Stroke Prevention Trial in Sickle Cell Anemia (STOP) is the first stroke prevention trial in Hb SS and the first randomized, controlled use of transfusion in Hb SS. This multi-center trial is designed to test whether reducing sickle hemoglobin to 30% or less with periodic blood transfusions will reduce first-time stroke by at least 70% compared to standard care. Primary endpoints will be clinically evident symptoms of cerebral infarction with consistent findings on Magnetic Resonance Imaging and Angiography (MRI/MRA) or symptomatic intracranial hemorrhage. Secondary endpoints will be asymptomatic brain lesions detected by MRI in brain areas not involved in primary endpoints. The design calls for a 6-month start-up interval, 18 months of TCD screening and randomization, and observation for stroke from entry through month 54. Key features of the trial are standardized TCD and MRI/MRA protocols interpreted blindly, and blinded adjudication of endpoints. The sample size (60 per treatment group) is based on prospective data relating TCD velocity to risk of stroke. A time-averaged mean velocity of greater than or equal to 200 cm/sec is associated with a 46% risk of cerebral infarction over 39 months. The sample size is sufficient to detect 70% reduction in the primary endpoint at 90% power. This trial will determine if transfusion is effective in the primary prevention of stroke. Secondary aims may further the understanding of the effects of transfusion on the brain and guide future research into cerebrovascular disease in Hb SS. (C) Elsevier Science Inc. 1998.