Department of Psychiatry and Behavioral Sciences, Stanford University, Stanford, California 94305, USA.
Biol Psychiatry. 2012 Jun 1;71(11):956-61. doi: 10.1016/j.biopsych.2012.01.014. Epub 2012 Feb 18.
An imbalance in the excitatory/inhibitory systems with abnormalities in the glutamatergic pathways has been implicated in the pathophysiology of autism. Furthermore, chronic redox imbalance was also recently linked to this disorder. The goal of this pilot study was to assess the feasibility of using oral N-acetylcysteine (NAC), a glutamatergic modulator and an antioxidant, in the treatment of behavioral disturbance in children with autism.
This was a 12-week, double-blind, randomized, placebo-controlled study of NAC in children with autistic disorder. Subjects randomized to NAC were initiated at 900 mg daily for 4 weeks, then 900 mg twice daily for 4 weeks and 900 mg three times daily for 4 weeks. The primary behavioral measure (Aberrant Behavior Checklist [ABC] irritability subscale) and safety measures were performed at baseline and 4, 8, and 12 weeks. Secondary measures included the ABC stereotypy subscale, Repetitive Behavior Scale-Revised, and Social Responsiveness Scale.
Thirty-three subjects (31 male subjects, 2 female subjects; aged 3.2-10.7 years) were randomized in the study. Follow-up data was available on 14 subjects in the NAC group and 15 in the placebo group. Oral NAC was well tolerated with limited side effects. Compared with placebo, NAC resulted in significant improvements on ABC irritability subscale (F = 6.80; p < .001; d = .96).
Data from this pilot investigation support the potential usefulness of NAC for treating irritability in children with autistic disorder. Large randomized controlled investigations are warranted.
兴奋性/抑制性系统失衡以及谷氨酸能通路异常与自闭症的病理生理学有关。此外，慢性氧化还原失衡也与这种疾病有关。本研究旨在评估口服 N-乙酰半胱氨酸（NAC），一种谷氨酸调节剂和抗氧化剂，治疗自闭症儿童行为障碍的可行性。
这是一项为期 12 周的双盲、随机、安慰剂对照研究，研究对象为自闭症儿童。随机分配到 NAC 组的患者首先每天服用 900mg，持续 4 周，然后每天服用 900mg 两次，持续 4 周，每天服用 900mg 三次，持续 4 周。主要行为测量（异常行为检查表[ABC]易激惹量表）和安全性测量在基线和第 4、8 和 12 周进行。次要测量包括 ABC 刻板行为量表、修订后的重复行为量表和社会反应量表。
共有 33 名患者（31 名男性，2 名女性；年龄 3.2-10.7 岁）被随机分为研究组。NAC 组和安慰剂组各有 14 名和 15 名患者有随访数据。口服 NAC 耐受性良好，副作用有限。与安慰剂相比，NAC 显著改善了 ABC 易激惹量表（F = 6.80；p <.001；d =.96）。
这项初步研究的结果支持 NAC 治疗自闭症儿童易激惹的潜在有效性。需要进行更大规模的随机对照研究。
