SILENT CEREBRAL INFARCT MULTI-CENTER CLINICAL TRIAL

无症状脑梗死多中心临床试验

• 批准号: 7603414
• 负责人: Allison A King
• 金额: $ 0.05万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-04-01 至 2007-09-16
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7603414
• 关键词: 13 year old; Accounting; Affect; Benefits and Risks; Blood Transfusion; Caring; Cerebrum; Child; Cognitive; Computer Retrieval of Information on Scientific Projects Database; Development; Drops; Enrollment; Ensure; Funding; Grant; Hemoglobin; Infarction; Institution; Intervention; Lead; Magnetic Resonance Imaging; Measurement; Multi-Institutional Clinical Trial; Outcome; Randomized; Randomized Controlled Clinical Trials; Rate; Research; Research Personnel; Resources; Risk; Sample Size; Screening procedure; Sickle Cell Anemia; Source; Standards of Weights and Measures; Stroke; Testing; United States National Institutes of Health; nervous system disorder; prophylactic

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Silent Cerebral Infarct (SCI) is the most common cause of severe neurological disease in children with sickle cell disease (SCD). This is a multi-center, randomized trial to test whether prophylactic blood transfusion therapy in children with SCI and SCD reduces the rate of subsequent overt strokes or new SCI, as defined by MRI. Of approximately 3,020 6-13 year old children with SCD eligible for screening, 1,880 will be asked to undergo MRIs. We estimate that 20% (376) will have SCI and that 10% (38) of these will be ineligible because of an elevated TCD measurement (200 cm/sec). We anticipate that 60% (203) of the remaining 338 will be randomized to either observation or blood transfusion therapy to keep the hemoglobin-S concentration less than 30%. This sample size ensures 90% power to detect an 86% reduction in new SCI, after accounting for 10% drop-out and 20% crossover rates. Intervention lasts for 36 months and each subject will receive a MRI and TCD at 12 months and MRI and cognitive assessment at study exit (36 months after enrollment). The primary outcome is development of stroke or new SCI. Secondary aims include determining whether: 1) prophylactic blood transfusion therapy limits further decline in intellectual abilities; and 2) the overall benefits of blood transfusion therapy for SCI outweigh risks associated with this therapy in a formal risk-benefit analysis. We anticipate results of this study could lead to a change in standard care practices for children affected with both SCD and SCI.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目及 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 无症状性脑梗死 (SCI) 是镰状细胞病 (SCD) 儿童严重神经系统疾病的最常见原因。 这是一项多中心、随机试验，旨在测试 SCI 和 SCD 儿童的预防性输血治疗是否可以降低 MRI 定义的后续明显中风或新发 SCI 的发生率。 在约 3,020 名 6-13 岁患有 SCD 且符合筛查条件的儿童中，其中 1,880 名将被要求接受 MRI 检查。 我们估计 20% (376) 人将患有 SCI，其中 10% (38) 人由于 TCD 测量值升高（200 厘米/秒）而不符合资格。 我们预计剩余 338 名患者中的 60% (203) 将被随机分配接受观察或输血治疗，以将血红蛋白-S 浓度保持在 30% 以下。 在考虑了 10% 的退出率和 20% 的交叉率后，这个样本量确保了 90% 的功效能够检测到新 SCI 减少 86%。 干预持续 36 个月，每位受试者将在 12 个月时接受 MRI 和 TCD，并在研究退出时（入组后 36 个月）接受 MRI 和认知评估。 主要结局是发生中风或新的 SCI。 次要目标包括确定：1）预防性输血疗法是否限制智力的进一步下降； 2) 在正式的风险效益分析中，SCI 输血治疗的总体益处超过了与该治疗相关的风险。 我们预计这项研究的结果可能会改变受 SCD 和 SCI 影响的儿童的标准护理实践。