White Matter Tract Changes Associated with Clinical Improvement in an Open-Label Trial Assessing Autologous Umbilical Cord Blood for Treatment of Young Children with Autism
White Matter Tract Changes Associated with Clinical Improvement in an Open-Label Trial Assessing Autologous Umbilical Cord Blood for Treatment of Young Children with Autism
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DOI：
10.1002/sctm.18-0251
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发表时间：
2019-02-01
影响因子：
6
通讯作者：
Dawson, Geraldine
中科院分区：
文献类型：
--
作者：
Carpenter, Kimberly L. H.;Major, Samantha;Dawson, Geraldine
自闭症谱系障碍（ASD）是一种异质性神经发育障碍，其特征是社会沟通障碍，存在有限的兴趣和重复行为。我们以前曾报道过在一项I期开放标签试验中单次输注自体脐带血治疗后，ASD儿童的行为得到显著改善，包括社会功能增强，沟通能力提高，临床症状减轻。在目前的研究中，我们的目的是了解这些改善是否与大脑结构连接的同时变化有关。25名2至6岁的ASD儿童参加了本试验。临床结果测量包括Vineland适应行为量表-II社会化子量表、表达性单字图片词汇测试-4和临床总体印象改善量表。在基线和6个月时，对19名儿童进行了25方向弥散张量成像和确定性纤维束成像。行为改善与额叶、颞叶和皮质下区域（海马和基底神经节）的白色连接性增加相关，这些区域先前已被证明在ASD中显示解剖学、连接性和功能异常。目前的结果表明，ASD儿童在自体脐带血输注治疗后社交技能的改善和症状的减轻与支持社交，沟通和语言能力的大脑网络的结构连接性增加有关。干细胞转化医学2019;8：138&10
Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by social communication deficits and the presence of restricted interests and repetitive behaviors. We have previously reported significant improvements in behavior, including increased social functioning, improved communication abilities, and decreased clinical symptoms in children with ASD, following treatment with a single infusion of autologous cord blood in a phase I open-label trial. In the current study, we aimed to understand whether these improvements were associated with concurrent changes in brain structural connectivity. Twenty-five 2- to 6-year-old children with ASD participated in this trial. Clinical outcome measures included the Vineland Adaptive Behavior Scales-II Socialization Subscale, Expressive One-Word Picture Vocabulary Test-4, and the Clinical Global Impression-Improvement Scale. Structural connectivity was measured at baseline and at 6 months in a subset of 19 children with 25-direction diffusion tensor imaging and deterministic tractography. Behavioral improvements were associated with increased white matter connectivity in frontal, temporal, and subcortical regions (hippocampus and basal ganglia) that have been previously shown to show anatomical, connectivity, and functional abnormalities in ASD. The current results suggest that improvements in social communication skills and a reduction in symptoms in children with ASD following treatment with autologous cord blood infusion were associated with increased structural connectivity in brain networks supporting social, communication, and language abilities. Stem Cells Translational Medicine 2019;8:138&10