TY - JOUR
T1 - 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
AU - Dawson, Geraldine
AU - Sun, Jessica M.
AU - Baker, Jennifer
AU - Carpenter, Kimberly
AU - Compton, Scott
AU - Deaver, Megan
AU - Franz, Lauren
AU - Heilbron, Nicole
AU - Herold, Brianna
AU - Horrigan, Joseph
AU - Howard, Jill
AU - Kosinski, Andrzej
AU - Major, Samantha
AU - Murias, Michael
AU - Page, Kristin
AU - Prasad, Vinod K.
AU - Sabatos-DeVito, Maura
AU - Sanfilippo, Fred
AU - Sikich, Linmarie
AU - Simmons, Ryan
AU - Song, Allen
AU - Vermeer, Saritha
AU - Waters-Pick, Barbara
AU - Troy, Jesse
AU - Kurtzberg, Joanne
N1 - Funding Information:
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.
Funding Information:
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.
Publisher Copyright:
© 2020 Elsevier Inc.
PY - 2020/7
Y1 - 2020/7
N2 - 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.
AB - 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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U2 - 10.1016/j.jpeds.2020.03.011
DO - 10.1016/j.jpeds.2020.03.011
M3 - Article
C2 - 32444220
AN - SCOPUS:85085763011
VL - 222
SP - 164-173.e5
JO - Journal of Pediatrics
JF - Journal of Pediatrics
SN - 0022-3476
ER -