A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder
A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder
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DOI：
10.1016/j.jpeds.2020.03.01
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发表时间：
2020-07-01
影响因子：
5.1
通讯作者：
Kurtzberg, Joanne
中科院分区：
文献类型：
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作者：
Dawson, Geraldine;Sun, Jessica M.;Kurtzberg, Joanne
目的评价脐带血输注对自闭症谱系障碍（ASD）患儿社交和沟通能力的改善是否安全及相关。这项前瞻性、随机、安慰剂对照、双盲研究纳入了180名年龄在2-7岁的ASD儿童，他们接受了单静脉自体（n = 56）或异体（n = 63） CB输注与安慰剂（n = 61），并在输注后6个月进行评估。结果CB输注安全，耐受性好。对整个样本的分析显示，没有证据表明CB与主要结果、社会沟通（Vineland适应行为量表[VABS-3]社会化领域）或次要结果、自闭症症状（广泛性发育障碍行为量表）和词汇（表达性单字图片词汇测试）的改善有关。也没有全面的证据表明不同类型的CB注入有不同的效果。在非智力残疾儿童（ID）的亚分析中，同种异体而非自体，在临床评定的临床总体印象改善量表中，CB与更大比例的儿童的改善相关，但改善的OR并不显著。接受CB治疗的无ID儿童在沟通技能（VABS-3沟通域）和探索性测量（包括对玩具的注意力和持续注意力（眼动追踪））以及α和β脑电图功率方面均有显着改善。总的来说，单次注射CB与改善社交技能或减轻自闭症症状无关。还需要更多的研究来确定CB输注对某些ASD儿童是否有效。
Objective To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD).Study design This prospective, randomized, placebo-controlled, double-blind study included 180 children with ASD, aged 2-7 years, who received a single intravenous autologous (n = 56) or allogeneic (n = 63) CB infusion vs placebo (n = 61) and were evaluated at 6 months postinfusion.Results CB infusion was safe and well tolerated. Analysis of the entire sample showed no evidence that CB was associated with improvements in the primary outcome, social communication (Vineland Adaptive Behavior Scales3 [VABS-3] Socialization Domain), or the secondary outcomes, autism symptoms (Pervasive Developmental Disorder Behavior Inventory) and vocabulary (Expressive One-Word Picture Vocabulary Test). There was also no overall evidence of differential effects by type of CB infused. In a subanalysis of children without intellectual disability (ID), allogeneic, but not autologous, CB was associated with improvement in a larger percentage of children on the clinician-rated Clinical Global Impression-Improvement scale, but the OR for improvement was not significant. Children without ID treated with CB showed significant improvements in communication skills (VABS-3 Communication Domain), and exploratory measures including attention to toys and sustained attention (eye-tracking) and increased alpha and beta electroencephalographic power.Conclusions Overall, a single infusion of CB was not associated with improved socialization skills or reduced autism symptoms. More research is warranted to determine whether CB infusion is an effective treatment for some children with ASD.