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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.JPed

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.

Authors+Show Affiliations

Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC. Electronic address: geraldine.dawson@duke.edu.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Emory University School of Medicine, Atlanta, GA.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC; Duke Brain Imaging and Analysis Center, Duke University School of Medicine, Durham, NC.Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32444220

Citation

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." The Journal of Pediatrics, vol. 222, 2020, pp. 164-173.e5.
Dawson G, Sun JM, Baker J, 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. 2020;222:164-173.e5.
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. The Journal of Pediatrics, 222, 164-e5. https://doi.org/10.1016/j.jpeds.2020.03.011
Dawson G, 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. 2020;222:164-173.e5. PubMed PMID: 32444220.
* Article titles in AMA citation format should be in sentence-case
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, Y1 - 2020/05/19/ PY - 2019/12/12/received PY - 2020/03/05/revised PY - 2020/03/05/accepted PY - 2020/5/24/pubmed PY - 2020/5/24/medline PY - 2020/5/24/entrez SP - 164 EP - 173.e5 JF - The Journal of pediatrics JO - J. Pediatr. VL - 222 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. SN - 1097-6833 UR - https://www.unboundmedicine.com/medline/citation/32444220/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_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0022-3476(20)30334-6 DB - PRIME DP - Unbound Medicine ER -
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