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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.
Dawson, Geraldine; Sun, Jessica M; Baker, Jennifer; Carpenter, Kimberly; Compton, Scott; Deaver, Megan; Franz, Lauren; Heilbron, Nicole; Herold, Brianna; Horrigan, Joseph; Howard, Jill; Kosinski, Andrzej; Major, Samantha; Murias, Michael; Page, Kristin; Prasad, Vinod K; Sabatos-DeVito, Maura; Sanfilippo, Fred; Sikich, Linmarie; Simmons, Ryan; Song, Allen; Vermeer, Saritha; Waters-Pick, Barbara; Troy, Jesse; Kurtzberg, Joanne.
Afiliación
  • Dawson G; 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.
  • Sun JM; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Baker J; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Carpenter K; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Compton S; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Deaver M; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Franz L; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Heilbron N; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Herold B; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Horrigan J; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Howard J; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Kosinski A; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Major S; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Murias M; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Page K; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Prasad VK; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Sabatos-DeVito M; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Sanfilippo F; Emory University School of Medicine, Atlanta, GA.
  • Sikich L; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Simmons R; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Song A; 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.
  • Vermeer S; Department of Psychiatry and Behavioral Sciences, Duke Center for Autism and Brain Development, Duke University School of Medicine, Durham, NC.
  • Waters-Pick B; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Troy J; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
  • Kurtzberg J; Marcus Center for Cellular Cures, Duke University School of Medicine, Durham, NC.
J Pediatr ; 222: 164-173.e5, 2020 07.
Article en En | MEDLINE | ID: mdl-32444220
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.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transfusión Sanguínea / Comunicación / Sangre Fetal / Trastorno del Espectro Autista Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Pediatr Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transfusión Sanguínea / Comunicación / Sangre Fetal / Trastorno del Espectro Autista Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Pediatr Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos