SILENT INFARCT TRANSFUSION TRIAL

无声梗塞输血试验

• 批准号: 7375113
• 负责人: Julie A Panepinto
• 金额: $ 0.26万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7375113
• 关键词: SILENT; INFARCT; TRANSFUSION; TRIAL

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. The goal of this trial is to determine whether blood transfusion therapy limits strokes and/or new or progressive MRI lesions in children with pre-existing silent cerebral infarcts, while secondary objectives examine the effectiveness of this therapy to limit decline of intellectual ability and establish a risk-benefit ratio. In this multicenter randomized study with participants from 22 clinical sites, 110 children from this site will be screened over the course of 3 years. Children, age 6-12 years with sickle cell anemia or beta-thalassemia, identified through the clinical database, will be screened with a brain MRI, complete phenotypic/demographic and quality of life questionnaires, and provide a blood sample for genetic testing. Those whose brain MRIs show evidence of a silent stroke will be examined by transcranial Doppler (TCD) and undergo neurological examination by a neurologist. 10 children whose TCD and neurology exams are within normal limits will have a repeat MRI performed, followed by randomization to receive either monthly blood transfusions (n = 5) or 3 years of observational care. All patients will undergo physical exams, laboratory testing, neurological and neurocognitive testing, 2 educational sessions on sickle cell disease and silent stroke, and risk-benefit analysis. The exit evaluation includes MRI, neurology examination, and cognitive testing, while repeat TCD and cognitive assessment will be done at 12-18 months for safety and interim analysis. GCRC nursing is requested for phlebotomy and red cell preparation, and space is requested to accommodate the children during transfusions. Informatics is asked to create scannable forms for the questionnaire, provide the use of scanning equipment, and create a database for the responses. The GCRC is also requested to cover costs for blood draw and cross-match for each transfusion, along with the packed red cells for transfusion.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构适用于该中心，这不一定是调查员的机构。该试验的目的是确定输血疗法限制了先前存在的无声大脑内感染儿童的中风和/或新的或进行性的MRI病变，而次要目标检查该治疗的有效性以限制智力能力的下降并建立风险比率。在这项与来自22个临床部位的参与者的多中心随机研究中，该站点的110名儿童将在3年的时间内进行筛查。通过临床数据库鉴定的镰状细胞贫血或β-杜比糖中贫血的6-12岁的儿童将通过脑部MRI进行筛查，完整的表型/人口统计学和生活质量问卷，并为基因测试提供血液样本。那些大脑MRIS显示出沉默中风的证据的人将通过经颅多普勒（TCD）检查并接受神经科医生的神经系统检查。 10名TCD和神经病学检查在正常范围内的儿童将重复进行MRI，然后随机分组以接受每月输血（n = 5）或3年观察护理。所有患者将接受体格检查，实验室检查，神经学和神经认知测试，2个有关镰状细胞病和无声中风的教育课程以及风险效益分析。退出评估包括MRI，神经病学检查和认知测试，而重复进行TCD和认知评估将在12-18个月进行安全和临时分析。要求GCRC护理进行静脉切开术和红细胞制备，并要求空间在输血期间容纳儿童。要求信息学为问卷创建可扫描表格，提供扫描设备的使用，并为回答创建数据库。还要求GCRC支付每次输血的血液抽血和交叉匹配成本，以及包装的红细胞进行输血。