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Stem Cells Transl Med. 2019 Feb;8(2):138-147. doi: 10.1002/sctm.18-0251. Epub 2019 Jan 8.

White Matter Tract Changes Associated with Clinical Improvement in an Open-Label Trial Assessing Autologous Umbilical Cord Blood for Treatment of Young Children with Autism.

Author information

1
Duke Center for Autism and Brain Development, Department of Psychiatry and Behavioral Sciences, Duke University School of Medicine, Durham, North Carolina, USA.
2
Brain Imaging and Analysis Center, Duke University Medical Center, Duke University School of Medicine, Durham, North Carolina, USA.
3
Duke Global Health Institute, Duke University School of Medicine, Duke University, Durham, North Carolina, USA.
4
Marcus Center for Cellular Cures, Duke University Medical Center, Duke University School of Medicine, Durham, North Carolina, USA.

Abstract

Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by social communication deficits and the presence of restricted interests and repetitive behaviors. We have previously reported significant improvements in behavior, including increased social functioning, improved communication abilities, and decreased clinical symptoms in children with ASD, following treatment with a single infusion of autologous cord blood in a phase I open-label trial. In the current study, we aimed to understand whether these improvements were associated with concurrent changes in brain structural connectivity. Twenty-five 2- to 6-year-old children with ASD participated in this trial. Clinical outcome measures included the Vineland Adaptive Behavior Scales-II Socialization Subscale, Expressive One-Word Picture Vocabulary Test-4, and the Clinical Global Impression-Improvement Scale. Structural connectivity was measured at baseline and at 6 months in a subset of 19 children with 25-direction diffusion tensor imaging and deterministic tractography. Behavioral improvements were associated with increased white matter connectivity in frontal, temporal, and subcortical regions (hippocampus and basal ganglia) that have been previously shown to show anatomical, connectivity, and functional abnormalities in ASD. The current results suggest that improvements in social communication skills and a reduction in symptoms in children with ASD following treatment with autologous cord blood infusion were associated with increased structural connectivity in brain networks supporting social, communication, and language abilities. Stem Cells Translational Medicine 2019;8:138&10.

KEYWORDS:

Autism spectrum disorder; Autologous umbilical cord blood; Diffusion tensor imaging; White matter connectivity

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