Efficacy and Safety of Intravenous Immunoglobulin in Children with Autism Spectrum Disorder with Immune...

Autism spectrum disorder (ASD) is a neurodevelopmental disorder. Accumulating evidence indicates that immune inflammation and oxidative stress may be involved in the pathogenesis of ASD. Intravenous immunoglobulin (IVIG), a well-established immunomodulatory agent, has demonstrated potential in ameliorating neuroinflammatory and behavioral symptoms in neuroimmune disorders. This prospective, open-label, single-arm study aimed to evaluate the short-term efficacy and safety of IVIG in children with ASD who have peripheral blood immune dysregulation.

A total of 41 children with ASD (aged 3-8 years) were enrolled. Participants received IVIG at a dose of 1 g/kg per infusion 1 month apart, resulting in a cumulative dose of 2 g/kg. The Social Responsiveness Scale-2 (SRS-2) was used to assess symptom changes at baseline, 1 month, and 2 months, and adverse events were recorded.

The SRS-2 total score and the scores of the Social Cognition, Social Communication and Social Motivation domains were significantly reduced at 2 months after IVIG intervention (P < 0.05). In terms of safety, two participants presented with a transient low-grade fever, two participants experienced vomiting, and one participant exhibited abdominal distension and poor appetite. All adverse events (AEs) were classified as Common Terminology Criteria for Adverse Events (CTCAE) grade 1 and resolved spontaneously without medical intervention.

This exploratory single-arm open-label study demonstrated that IVIG is well tolerated and may be associated with improvements in social behavior among children with ASD with immune dysregulation. However, since the primary outcome measure (SRS-2) was caregiver-reported, response bias should be taken into consideration. As an exploratory and hypothesis-generating study, no definitive conclusions regarding therapeutic efficacy or causal relationships can be drawn from these findings. Therefore, its therapeutic efficacy remains inconclusive, and the results should be interpreted with caution. Further multicenter, double-blind controlled trials are warranted to confirm the efficacy of IVIG and explore the underlying mechanisms involved.

chictr.org.cn (registration number ChiCTR2500111846).