N-Acetylcysteine, autism, irritability

February 28, 2012

A Randomized Controlled Pilot Trial of Oral N-Acetylcysteine in Children

Hardan AY, Fung LK, Libove RA, Obukhanych TV, Nair S, Herzenberg LA,

Frazier TW, Tirouvanziam R.

Biol Psychiatry. 2012 Feb 17

BACKGROUND: 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.

METHODS: 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.

RESULTS: 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).

CONCLUSIONS: 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.

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