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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.

Publication ,  Journal Article
Dawson, G; Sun, JM; Baker, J; Carpenter, K; Compton, S; Deaver, M; Franz, L; Heilbron, N; Herold, B; Horrigan, J; Howard, J; Kosinski, A ...
Published in: J Pediatr
July 2020

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.

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Published In

J Pediatr

DOI

EISSN

1097-6833

Publication Date

July 2020

Volume

222

Start / End Page

164 / 173.e5

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Prospective Studies
  • Pediatrics
  • Male
  • Language Tests
  • Humans
  • Follow-Up Studies
  • Fetal Blood
  • Female
  • Double-Blind Method
 

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APA
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MLA
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Dawson, G., Sun, J. M., Baker, J., Carpenter, K., Compton, S., Deaver, M., … 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. J Pediatr, 222, 164-173.e5. https://doi.org/10.1016/j.jpeds.2020.03.011
Dawson, Geraldine, Jessica M. Sun, Jennifer Baker, Kimberly Carpenter, Scott Compton, Megan Deaver, Lauren Franz, et al. “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 222 (July 2020): 164-173.e5. https://doi.org/10.1016/j.jpeds.2020.03.011.
Dawson, Geraldine, et al. “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, vol. 222, July 2020, pp. 164-173.e5. Pubmed, doi:10.1016/j.jpeds.2020.03.011.
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. 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.
Journal cover image

Published In

J Pediatr

DOI

EISSN

1097-6833

Publication Date

July 2020

Volume

222

Start / End Page

164 / 173.e5

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Prospective Studies
  • Pediatrics
  • Male
  • Language Tests
  • Humans
  • Follow-Up Studies
  • Fetal Blood
  • Female
  • Double-Blind Method