Effects of Citicoline in Children with Autism Spectrum Disorder: A Randomized, Open-label Clinical Trial.

Purpose: Autism Spectrum Disorder (ASD) is a neurodevelopmental condition characterized by impaired social interactions, communication challenges, and repetitive behaviors. Citicoline, a precursor in phosphatidylcholine synthesis, has shown potential cognitive benefits in various neurological conditions. This study aimed to evaluate the effects of citicoline on cognitive and behavioral functions in patients with ASD.

Methods: A randomized, open-label, parallel-design clinical trial was conducted on 101 children with ASD (aged under 18 years) at Imam Reza Clinic and Namazi Hospital, Shiraz, Iran. Participants were divided into citicoline (n = 45) and control groups (n = 56). Citicoline (10 mg/kg) was administered intramuscularly. The Modified Checklist for Autism in Toddlers (M-CHAT) scale was used for children under 3 years, and the Gilliam Autism Rating Scale (GARS) for those over 3 years, to assess ASD symptoms before and after 2 months of treatment.

Results: At baseline, no significant differences were found between the citicoline and control groups in M-CHAT and GARS scores (P-value: 0.587, P-value: 0.100, respectively). After 2 months, there was no significant difference between the two groups either (P-value: 0.188, P-value: 0.269, respectively). The citicoline did not show any beneficial effects (compared to the control group) in any of the GARS subscales.

Conclusion: Citicoline did not provide significant clinical benefits for patients with ASD. Currently, there is not enough evidence to support the prescription of citicoline for children with ASD, and this practice should be discouraged unless other high-quality evidence shows the contrary.