Casella James F, King Allison A, Barton Bruce, White Desiree A, Noetzel Michael J, Ichord Rebecca N, Terrill Cindy, Hirtz Deborah, McKinstry Robert C, Strouse John J, Howard Thomas H, Coates Thomas D, Minniti Caterina P, Campbell Andrew D, Vendt Bruce A, Lehmann Harold, Debaun Michael R
Pediatric Hematology, Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA.
Pediatr Hematol Oncol. 2010 Mar;27(2):69-89. doi: 10.3109/08880010903360367.
Silent cerebral infarct (SCI) is the most common cause of serious neurological disease in sickle cell anemia (SCA), affecting approximately 22% of children. The goal of this trial is to determine whether blood transfusion therapy will reduce further neurological morbidity in children with SCI, and if so, the magnitude of this benefit.
The Silent Cerebral Infarct Transfusion (SIT) Trial includes 29 clinical sites and 3 subsites, a Clinical Coordinating Center, and a Statistical and Data Coordinating Center, to test the following hypothesis: prophylactic blood transfusion therapy in children with SCI will result in at least an 86% reduction in the rate of subsequent overt strokes or new or progressive cerebral infarcts as defined by magnetic resonance imaging (MRI) of the brain. The intervention is blood transfusion versus observation. Two hundred and four participants (102 in each treatment assignment) will ensure 85% power to detect the effect necessary to recommend transfusion therapy (86% reduction), after accounting for 10% drop out and 19% crossover rates. MRI examination of the brain is done at screening, immediately before randomization and study exit. Each randomly assigned participant receives a cognitive test battery at study entry, 12-18 months later, and study exit and an annual neurological examination. Blood is obtained from all screened participants for a biologic repository containing serum and a renewable source of DNA.
The SIT Trial could lead to a change in standard care practices for children affected with SCA and SCI, with a consequent reduction in neurological morbidity.
无症状脑梗死（SCI）是镰状细胞贫血（SCA）中严重神经疾病的最常见病因，约22%的儿童受其影响。本试验的目的是确定输血疗法是否会降低SCI患儿进一步的神经疾病发病率，若能降低，其益处的程度如何。
无症状脑梗死输血（SIT）试验包括29个临床站点和3个分站、一个临床协调中心以及一个统计和数据协调中心，以检验以下假设：SCI患儿的预防性输血疗法将使后续明显中风或由脑部磁共振成像（MRI）定义的新的或进展性脑梗死发生率至少降低86%。干预措施为输血与观察。在考虑10%的退出率和19%的交叉率后，204名参与者（每个治疗组102名）将确保有85%的把握度检测出推荐输血疗法所需的效果（降低86%）。在筛查时、随机分组前及研究结束时进行脑部MRI检查。每个随机分组的参与者在研究开始时、12 - 18个月后、研究结束时接受一套认知测试，并每年进行一次神经学检查。从所有筛查参与者采集血液，用于建立包含血清和可再生DNA来源的生物样本库。
SIT试验可能会改变SCA和SCI患儿的标准护理做法，从而降低神经疾病发病率。
