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.

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.

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Citation

Published Version (Please cite this version)

10.1002/sctm.18-0251

Publication Info

Carpenter, Kimberly LH, Samantha Major, Catherine Tallman, Lyon W Chen, Lauren Franz, Jessica Sun, Joanne Kurtzberg, Allen Song, et al. (2019). 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. Stem cells translational medicine, 8(2). pp. 138–147. 10.1002/sctm.18-0251 Retrieved from https://hdl.handle.net/10161/24581.

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Scholars@Duke

Carpenter

Kimberly Carpenter

Assistant Professor in Psychiatry and Behavioral Sciences

Dr. Kimberly Carpenter is a clinical neuroscientist specializing in understanding complex brain-behavior relationships in young children with autism and associated disorders. Her program of research includes four interrelated research themes: (1) Understanding the impact of comorbid disorders on clinical and behavioral outcomes of young autistic children; (2) Identification of early risk factors for the development of psychiatric and neurodevelopmental disorders; (3) Identification of brain-based biomarkers for group stratification and treatment response tracking in young children; and (4) Improving methods for screening, early identification, and treatment monitoring in autism and associated disorders. She currently leads an innovative research program exploring the shared and unique impacts that co-occurring anxiety and ADHD have on brain and behavioral biomarkers in young autistic children. She was the first to demonstrate that sensory over-responsivity, a symptom that has been described as part of a number of disorders including autism, anxiety, and ADHD, is a specific and unidirectional risk factor for the development of anxiety disorders in young children. She was also the first to demonstrate that, when accounting for comorbidity among individual anxiety disorders, specific anxiety disorders are associated with phenotypically meaningful differences in brain connectivity using MRI. Dr. Carpenter has also collaborated with experts in early childhood mental health, computer science, and engineering to develop novel technologies that utilize multi-modal methods via computer vision and machine learning to develop, refine, and test novel screening tools for early identification and treatment monitoring in young children with autism and related disorders.

Franz

Lauren Franz

Associate Professor of Psychiatry and Behavioral Sciences

Dr Franz's research focuses on improving access to evidence-based services and support for neurodevelopmental disabilities, including autism, in diverse, low-resource, local and global communities.

Jessica Muller Sun

Assistant Professor of Pediatrics

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