Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial.
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2017-12
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Cerebral palsy (CP) is a condition affecting young children that causes lifelong disabilities. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. After demonstrating safety, we conducted a phase II trial of autologous cord blood (ACB) infusion in children with CP to test whether ACB could improve function (ClinicalTrials.gov, NCT01147653; IND 14360). In this double-blind, placebo-controlled, crossover study of a single intravenous infusion of 1-5 × 107 total nucleated cells per kilogram of ACB, children ages 1 to 6 years with CP were randomly assigned to receive ACB or placebo at baseline, followed by the alternate infusion 1 year later. Motor function and magnetic resonance imaging brain connectivity studies were performed at baseline, 1, and 2 years post-treatment. The primary endpoint was change in motor function 1 year after baseline infusion. Additional analyses were performed at 2 years. Sixty-three children (median age 2.1 years) were randomized to treatment (n = 32) or placebo (n = 31) at baseline. Although there was no difference in mean change in Gross Motor Function Measure-66 (GMFM-66) scores at 1 year between placebo and treated groups, a dosing effect was identified. In an analysis 1 year post-ACB treatment, those who received doses ≥2 × 107 /kg demonstrated significantly greater increases in GMFM-66 scores above those predicted by age and severity, as well as in Peabody Developmental Motor Scales-2 Gross Motor Quotient scores and normalized brain connectivity. Results of this study suggest that appropriately dosed ACB infusion improves brain connectivity and gross motor function in young children with CP. Stem Cells Translational Medicine 2017;6:2071-2078.
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Sun, Jessica M, Allen W Song, Laura E Case, Mohamad A Mikati, Kathryn E Gustafson, Ryan Simmons, Ricki Goldstein, Jodi Petry, et al. (2017). Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem cells translational medicine, 6(12). pp. 2071–2078. 10.1002/sctm.17-0102 Retrieved from https://hdl.handle.net/10161/24589.
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Scholars@Duke
Jessica Muller Sun
Allen W Song
The research in our lab is concerned with advancing structural and functional MRI methodologies (e.g. fast and high-resolution imaging techniques) for human brain imaging. We also aim to improve our understanding of functional brain signals, including spatiotemporal characterizations of the blood oxygenation level dependent contrast and alternative contrast mechanisms that are more directly linked to the neuronal activities. Additional effort is invested in applying and validating the developed methods to study human functional neuroanatomy.
Kathryn E. Gustafson
My scholarly interests and expertise are in pediatric neurodevelopmental outcomes assessment and research as well as child and parent coping with chronic childhood illness. In the 1990s, I collaborated with Dr. Robert Thompson in investigating the transactional biopsychosocial model of adaptation to pediatric conditions in children and families. Our research program was funded by the NIH and culminated in the publication of our book, Adaptation to Chronic Childhood Illness. Since that time, I have worked closely with the Division of Neonatology and the Duke Neonatal-Perinatal Research Unit on neurodevelopmental outcomes research with high-risk infants, toddlers, and school-age children. I am a gold standard psychology consultant to the Neonatal Research Network (NRN) of the NIH/NICHD, train and certify psychologists nationally and internationally in infant and toddler developmental assessment for numerous research groups, and serve as consultant for protocol development. In addition, I collaborate with colleagues in Pediatric Ophthalmology to investigate preterm optic nerve anatomy assessed via optical coherence tomography imaging and the association with neurodevelopment. I am also involved in investigations of umbilical cord blood stem cell transplant for young children with hypoxic ischemic encephalopathy, cerebral palsy, and inborn errors of metabolism, such as Krabbe disease, with colleagues in the Pediatric Transplant and Cellular Therapy program.
Colleen A McLaughlin
Gordon Worley
Dr. Gordon Worley specializes in Neurodevelopmental Disabilities, a sub-specialty of Pediatrics focusing on the management of the medical problems of children with physical disabilities. The principal diagnoses he treats are cerebral palsy, spina bifida, and Down syndrome. HIs research pertains to clinical problems of children with these conditions. Current research interests include genetic factors influencing outcomes of Extremely Low Birth Weight Infants using analyses of a large data set; comparison of outcomes of prenatal (in utero) surgery vs postnatal for lesion closure in meningomyelocele using the National Spina Bifida Patient Registry; Down Syndrome Disintegrative Disorder; and associations of ADHD with high risk sexual behaviors using analyses of a large data set.
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