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 - 27/01/21
, Jessica M. Sun, MD 2, Jennifer Baker, RN 2, Kimberly Carpenter, PhD 1, Scott Compton, PhD 1, Megan Deaver, PhD 1, Lauren Franz, MB, ChB 1, Nicole Heilbron, PhD 1, Brianna Herold, MS 1, Joseph Horrigan, MD 1, Jill Howard, PhD 1, Andrzej Kosinski, PhD 2, Samantha Major, MS 1, Michael Murias, PhD 1, Kristin Page, MD 2, Vinod K. Prasad, MD 2, Maura Sabatos-DeVito, PhD 1, Fred Sanfilippo, MD 3, Linmarie Sikich, MD 1, Ryan Simmons, PhD 2, Allen Song, PhD 2, 4, Saritha Vermeer, PhD 1, Barbara Waters-Pick, MD 2, Jesse Troy, PhD 2, Joanne Kurtzberg, MD 2Abstract |
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.
Le texte complet de cet article est disponible en PDF.Abbreviations and Acronyms : AE, ASD, CB, CGI-I, CGI-S, CNS, DSM-5, EEG, EOWPVT, FDR, HLA, ID, IV, IND, MRI, NVIQ, PANDAS, PDDBI, SAE, SS, TNC, VABS-3
Plan
| Supported by The Marcus Foundation, Atlanta, GA. K.C. reports technology unrelated to the submitted work that has been licensed and has benefited financially, and has a patent 15141391 pending. G.D. reports personal fees from Janssen, Roche, and Akili, and technology unrelated to the submitted work that has been licensed and has benefited financially, has patents 62757234, 62757226, 15141391, and 62470431 pending. J. Horrrigan reports personal fees from AMO Pharma Ltd. J. Howard reports personal fees from Roche. J.K. has a patent 62470431 pending. F.S. is a paid consultant to The Marcus Foundation as Medical Director. L.S. receives personal fees from Neuren, Roche, and nonfinancial support from Neos Pharmaceuticals. J.S. has a patent 62470431 pending. J.T. reports personal fees from Cohortias, EMMES Corporation, Community Data Roundtable, AegisCN, Gamida Cell, and a patent 62470431 pending. The study sponsor was not involved in the study design; the collection, analysis, and interpretation of data; the writing of the report; or the decision to submit the paper for publication. The other authors declare no conflicts of interest. |
Vol 222
P. 164 - juillet 2020 Retour au numéro
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