A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder

Geraldine Dawson*, Jessica M. Sun, Jennifer Baker, Kimberly Carpenter, Scott Compton, Megan Deaver, Lauren Franz, Nicole Heilbron, Brianna Herold, Joseph Horrigan, Jill Howard, Andrzej Kosinski, Samantha Major, Michael Murias, Kristin Page, Vinod K. Prasad, Maura Sabatos-DeVito, Fred Sanfilippo, Linmarie Sikich, Ryan SimmonsAllen Song, Saritha Vermeer, Barbara Waters-Pick, Jesse Troy, Joanne Kurtzberg

*Corresponding author for this work

Research output: Contribution to journalArticle

1 Scopus citations

Abstract

Objective: To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD). Study design: This prospective, randomized, placebo-controlled, double-blind study included 180 children with ASD, aged 2-7 years, who received a single intravenous autologous (n = 56) or allogeneic (n = 63) CB infusion vs placebo (n = 61) and were evaluated at 6 months postinfusion. Results: CB infusion was safe and well tolerated. Analysis of the entire sample showed no evidence that CB was associated with improvements in the primary outcome, social communication (Vineland Adaptive Behavior Scales-3 [VABS-3] Socialization Domain), or the secondary outcomes, autism symptoms (Pervasive Developmental Disorder Behavior Inventory) and vocabulary (Expressive One-Word Picture Vocabulary Test). There was also no overall evidence of differential effects by type of CB infused. In a subanalysis of children without intellectual disability (ID), allogeneic, but not autologous, CB was associated with improvement in a larger percentage of children on the clinician-rated Clinical Global Impression-Improvement scale, but the OR for improvement was not significant. Children without ID treated with CB showed significant improvements in communication skills (VABS-3 Communication Domain), and exploratory measures including attention to toys and sustained attention (eye-tracking) and increased alpha and beta electroencephalographic power. Conclusions: Overall, a single infusion of CB was not associated with improved socialization skills or reduced autism symptoms. More research is warranted to determine whether CB infusion is an effective treatment for some children with ASD.

Original languageEnglish (US)
Pages (from-to)164-173.e5
JournalJournal of Pediatrics
Volume222
DOIs
StatePublished - Jul 2020
Externally publishedYes

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Fingerprint Dive into the research topics of 'A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder'. Together they form a unique fingerprint.

  • Cite this

    Dawson, G., Sun, J. M., Baker, J., Carpenter, K., Compton, S., Deaver, M., Franz, L., Heilbron, N., Herold, B., Horrigan, J., Howard, J., Kosinski, A., Major, S., Murias, M., Page, K., Prasad, V. K., Sabatos-DeVito, M., Sanfilippo, F., Sikich, L., ... Kurtzberg, J. (2020). A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder. Journal of Pediatrics, 222, 164-173.e5. https://doi.org/10.1016/j.jpeds.2020.03.011