Efficacy and safety of allogeneic umbilical cord blood mononuclear cells therapy in children with autism spectrum disorder and immune dysregulation: a single-center, double-blinded, randomized, placebo-controlled trial.
Zhang Z, Wang G, Han F, Zhou H, Liang Y et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001
This study tested whether umbilical cord blood cells given by injection could help children with autism spectrum disorder who also have immune system problems. Thirty-four children aged 3 to 8 years received either the cell treatment or a placebo four times over four weeks, while continuing their usual therapy. The treated group showed modest improvements in social responsiveness compared to placebo, with no serious safety problems reported.
Key points
Children receiving umbilical cord blood mononuclear cells showed significantly greater reduction in Social Responsiveness Scale-2 total scores compared to placebo (difference of -6.03 points, P = 0.045)
The cell therapy produced the largest effect on the Social Cognition domain (difference of -9.95 points, P = 0.002)
No major adverse events related to the cell transplantation were reported
The study involved 34 children aged 3 to 8 years with autism spectrum disorder and immune dysregulation
Treatment consisted of four intravenous infusions of 3 × 10⁸ cells administered one week apart
Limitations
This is a single-center trial with a small sample size (34 children), which limits generalizability. The improvements shown are modest and it remains unclear whether these changes would be clinically meaningful for children and families.
Abstract
Background Immune-inflammatory dysregulation is increasingly implicated in the pathogenesis of autism spectrum disorder (ASD). Umbilical cord blood mononuclear cells (UCB-MNCs), with their immunomodulatory effects, represent a promising therapeutic avenue by potentially alleviating neuroinflammation. This study aimed to evaluate the safety and efficacy of multiple intravenous infusions of allogeneic UCB-MNCs in children with ASD and peripheral immune dysregulation. Methods In this single-center, double-blind, randomized, placebo-controlled trial (RCT), 34 children (aged 3 to 8 years) diagnosed with ASD were randomly assigned to receive intravenous infusion of allogeneic UCB-MNCs (3 × 10⁸ cells per infusion) (n = 17) or placebo (n = 17) four times with 1-week interval, alongside their existing rehabilitation therapy, and followed up for 13 weeks. The primary endpoint were the Total score of the Social Responsiveness Scale-2 (SRS-2). Additional endpoints was changes of the five domains of the SRS-2, the Vineland Adaptive Behavior Scales-3 (Vineland-3), the Childhood Autism Rating Scale (CARS), Aberrant Behavior Checklist (ABC), Swanson, Nolan, and Pelham Rating Scale-IV (SNAP-IV), Self-Rating Anxiety Scale (SAS) and safety. Results At final follow-up, the UCB-MNCs group demonstrated a significantly greater reduction in the SRS-2 Total scores (LS Mean Difference = - 6.03, 95% confidence interval [CI]: - 11.93 to - 0.14, P = 0.045) and Social Cognition domain (LS Mean Difference = - 9.95, 95% confidence interval [CI]: - 15.90 to - 4.00, P = 0.002) scores compared to the placebo group. No major allogeneic UCB-MNCs transplantation-related adverse events occurred. Conclusion Allogeneic UCB-MNCs transplantation demonstrated preliminary safety and potential efficacy in improving specific aspects of symptoms in children with ASD and peripheral immune dysregulation. Trial registration This study was registered on ChiCTR.org.cn (Identifier: ChiCTR2400082762). Date registered 12 April 2024. https://www.chictr.org.cn/index.html .
Findings the Hub has extracted
Each claim below is labelled by its type of design and links back to this report.
Multiple intravenous infusions of allogeneic umbilical cord blood mononuclear cells (3 × 10⁸ cells per infusion, four infusions at weekly intervals)
Population
34 children aged 3 to 8 years diagnosed with autism spectrum disorder and peripheral immune dysregulation
Key finding
UCB-MNCs group showed significantly greater reduction in Social Responsiveness Scale-2 total scores (LS Mean Difference = -6.03, 95% CI: -11.93 to -0.14, P = 0.045)
