SILENT INFARCT TRANSFUSION TRIAL

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。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。本试验的目的是确定输血治疗是否限制了既存无症状性脑梗死儿童的卒中和/或新发或进行性MRI病变，而次要目的是检查该治疗限制智力下降的有效性并建立风险-获益比。在这项多中心随机研究中，参与者来自22个临床研究中心，110名儿童将在3年内接受筛选。通过临床数据库确定的6-12岁镰状细胞性贫血或β地中海贫血儿童将接受脑部MRI筛查，完成表型/人口统计学和生活质量问卷调查，并提供血液样本进行基因检测。那些脑部核磁共振成像显示无症状中风证据的人将接受经颅多普勒（TCD）检查，并接受神经科医生的神经系统检查。10名TCD和神经学检查在正常范围内的儿童将接受重复MRI检查，然后随机接受每月输血（n = 5）或3年观察性护理。所有患者都将接受体格检查、实验室检查、神经和神经认知测试、2次关于镰状细胞病和无症状性卒中的教育课程以及风险获益分析。退出评估包括MRI、神经学检查和认知测试，而重复TCD和认知评估将在12-18个月进行安全性和中期分析。静脉切开术和红细胞制备需要GCRC护理，输血期间需要空间容纳儿童。信息学被要求为调查表创建可扫描的表格，提供扫描设备的使用，并为答复创建数据库。还要求GCRC支付每次输血的抽血和交叉配血费用，沿着输血用浓缩红细胞的费用。

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.