Autism spectrum disorders (ASD) are influenced by interacting maternal and environmental risk factors. High-dose folinic acid has shown improvement in verbal communication in ASD children. The EFFET randomized placebo-controlled trial (NCT02551380) aimed to evaluate the efficacy of folinic acid (FOLINORAL®) at a lower dose of 5 mg twice daily. Nineteen children were included in the EFFET trial. The primary efficacy outcome was improvement of Autism Diagnostic Observation Schedule (ADOS) score. The secondary outcomes were the improvement in ADOS sub scores communication, social interactions, Social Responsiveness Score (SRS) and treatment safety. The global ADOS score and social interaction and communication sub scores were significantly improved at week 12 compared to baseline in the folinic acid group (P = 0.003, P = 0.004 and P = 0.022, respectively), but not in the placebo group (P = 0.574, P = 0.780, P = 0.269, respectively). We observed a greater change of ADOS global score (-2.78 vs. -0.4 points) and (-1.78 vs. 0.20 points) in the folinic acid group, compared to the placebo group. No serious adverse events were observed. This pilot study showed significant efficacy of folinic acid with an oral formulation that is readily available. It opens a perspective of therapeutic intervention with folinic acid but needs to be confirmed by a multi-center trial on a larger number of children.

中文翻译：

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在EFFET安慰剂对照的随机试验中，亚叶酸可改善自闭症的评分。

自闭症谱系障碍（ASD）受孕产妇和环境危险因素相互作用的影响。大剂量亚叶酸已显示出ASD儿童的言语交流得到改善。EFFET随机安慰剂对照试验（NCT02551380）旨在评估每天两次低剂量5 mg的亚叶酸（FOLINORAL®）的疗效。EFFET试验中包括19名儿童。主要疗效结果是自闭症诊断观察时间表（ADOS）评分的改善。次要结果是改善ADOS子评分沟通，社交互动，社交反应评分（SRS）和治疗安全性。与亚叶酸组的基线相比，第12周的总体ADOS得分以及社交互动和沟通子得分显着提高（P = 0.003，P = 0.004和P = 0.022，），但在安慰剂组中则没有（分别为P = 0.574，P = 0.780，P = 0.269）。我们观察到，与安慰剂组相比，亚叶酸组的ADOS总体得分（-2.78 vs. -0.4分）和（-1.78 vs.0.20分）变化更大。没有观察到严重的不良事件。这项先导研究表明，亚叶酸与口服制剂的有效性相当高，可立即获得。它为亚叶酸的治疗干预开辟了前景，但需要通过对更多儿童进行的多中心试验来证实。这项先导研究表明，亚叶酸与口服制剂的有效性相当高，可立即获得。它为亚叶酸的治疗干预开辟了前景，但需要通过对更多儿童进行的多中心试验来证实。这项先导研究表明，亚叶酸与口服制剂的显着疗效均很容易获得。它为亚叶酸的治疗干预开辟了前景，但需要通过对更多儿童进行的多中心试验来证实。