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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. J Pediatr 2020 Jul;222:164-173.e5



Pubmed ID




Scopus ID

2-s2.0-85085763011 (requires institutional sign-in at Scopus site)   31 Citations


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.

Author List

Dawson G, Sun JM, 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 VK, Sabatos-DeVito M, Sanfilippo F, Sikich L, Simmons R, Song A, Vermeer S, Waters-Pick B, Troy J, Kurtzberg J


Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Autism Spectrum Disorder
Blood Transfusion
Child, Preschool
Double-Blind Method
Fetal Blood
Follow-Up Studies
Language Tests
Prospective Studies
Treatment Outcome