Scholarlink

Introduction <p>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.</p> Methods <p>A total of 41 children with ASD (aged 3–8&#xa0;years) were enrolled. Participants received IVIG at a dose of 1&#xa0;g/kg per infusion 1 month apart, resulting in a cumulative dose of 2&#xa0;g/kg. The Social Responsiveness Scale-2 (SRS-2) was used to assess symptom changes at baseline, 1&#xa0;month, and 2&#xa0;months, and adverse events were recorded.</p> Results <p>The SRS-2 total score and the scores of the Social Cognition, Social Communication and Social Motivation domains were significantly reduced at 2&#xa0;months after IVIG intervention (<i>P</i> &lt; 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.</p> Conclusion <p>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.</p> Trial Registration <p>chictr.org.cn (registration number ChiCTR2500111846).</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Efficacy and Safety of Intravenous Immunoglobulin in Children with Autism Spectrum Disorder with Immune Dysregulation: A Prospective, Open-Label, Single-Arm Study

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年3月19日

查看全文
  • Ziyan Zhang,
  • Li Zhang,
  • Gang Zhu,
  • Yan Liang,
  • Ping Pang,
  • Huimin Yan,
  • Xinyu Cao,
  • Jing Wang,
  • Xiuyu Shi,
  • Linyan Hu,
  • Guang Yang

摘要

Introduction

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.

Methods

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.

Results

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.

Conclusion

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.

Trial Registration

chictr.org.cn (registration number ChiCTR2500111846).