自体脐带血与个体化补充治疗自闭症谱系障碍:CORDUS研究结果。

Felician A Stancioiu, Raluca Bogdan, Bogdan Ivanescu, Radu Dumitrescu
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引用次数: 0

摘要

背景:细胞疗法在自闭症谱系障碍(autism spectrum disorder, ASD)中开启了一个重要的治疗新方向,而ASD病理生理的丰富多样性和不同类型的细胞疗法也促使人们在临床研究ASD病因和细胞疗法方面付出了同样多的努力。到目前为止,干细胞在临床试验中产生的结果好坏参半,在患者水平上,结果从令人印象深刻到没有改善。在这种情况下,我们对ASD儿童进行了自体脐带血(ACB)和一种非安慰剂的物质干预,即个体化的补充组合(ICS)。目的:比较ACB与ICS的疗效,寻找与患儿病情进展相关的标志物,以便更好地预测ACB的疗效。方法:CORDUS临床研究是一项交叉研究,对56名儿童依次给予口服ICS和静脉注射ACB;ACB作为一项住院治疗。治疗前和治疗后6个月由独立心理治疗师评估治疗效果,采用《自闭症治疗评估表》、《幼儿自闭症定量表》和16项对照表评分。在每次干预之前和之后,参与者都进行了一组血液测试,包括炎症、代谢和氧化标志物,以及神经元特异性烯醇化酶。结果:在脐带血或补品使用期间和之后均未发现严重不良反应。ACB改善了78%的3-7岁儿童的评估分数(n = 28),但对8岁以上或体重大于35 kg的儿童的效果要差得多(n = 28;只有11%的孩子成绩有所提高)。28例患儿中,5例患儿ACB疗效优于ACB, 23例患儿ACB疗效优于ICS (P < 0.05);高水平的炎症和铁蛋白没有改善。在儿童的进步中发现了大量的个体差异,在统计上,ACB后在语言表达和社会互动等方面有显著的改善,但在易怒或攻击行为方面没有显著改善。结论:ACB治疗ASD疗效优于ICS;高炎症、高铁蛋白、高年龄和高体重预示改善不大;ACB治疗ASD的疗效有待更多的临床研究。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Autologous cord blood vs individualized supplements in autistic spectrum disorder: CORDUS study results.

Background: Cellular therapies have started an important new therapeutic direction in autistic spectrum disorder (ASD), and the ample diversity of ASD pathophysiology and the different types of cell therapies prompt an equally ample effort to employ clinical studies for studying the ASD causes and cell therapies. Stem cells have yielded so far mixed results in clinical trials, and at patient level the results varied from impressive to no improvement. In this context we have administered autologous cord blood (ACB) and a non-placebo, material intervention represented by an individualized combination of supplements (ICS) to ASD children.

Aim: To compare the efficacy of ACB vs ICS and find markers correlated with the child's progress in order to better predict ACB efficacy.

Methods: CORDUS clinical study is a crossover study in which both oral ICS and intravenous ACB were sequentially administered to 56 children; ACB was infused as an inpatient procedure. Treatment efficacy was evaluated pre-treatment and post-treatment at 6 months by an independent psychotherapist with Autism Treatment Evaluation Checklist, Quantitative Checklist for Autism in Toddlers and a 16-item comparative table score, after interviewing the children's parents and therapists. Before and after each intervention participants had a set of blood tests including inflammatory, metabolic and oxidative markers, and the neuronal specific enolase.

Results: No serious adverse reactions were noted during and after cord blood or supplement administration. ACB improved evaluation scores in 78% of children with age 3-7-years (n = 28), but was much less effective in kids older than 8 years or with body weight of more than 35 kg (n = 28; only 11% of children improved scores). ICS yielded better results than ACB in 5 cases out of 28, while in 23 kids ACB brought more improvement than ICS (P < 0.05); high initial levels of inflammation and ferritin were associated with no improvement. Ample individual differences were noted in children's progress, and statistically significant improvements were seen after ACB on areas such as verbalization and social interaction, but not on irritability or aggressive behavior.

Conclusion: ACB has superior efficacy to ICS in ASD; high inflammation, ferritin, age and body weight predict less improvement; more clinical studies are needed for studying ACB efficacy in ASD.

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