z-logo
open-access-imgOpen Access
Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo‐Controlled Trial
Author(s) -
Sun Jessica M.,
Song Allen W.,
Case Laura E.,
Mikati Mohamad A.,
Gustafson Kathryn E.,
Simmons Ryan,
Goldstein Ricki,
Petry Jodi,
McLaughlin Colleen,
WatersPick Barbara,
Chen Lyon W.,
Wease Stephen,
Blackwell Beth,
Worley Gordon,
Troy Jesse,
Kurtzberg Joanne
Publication year - 2017
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.1002/sctm.17-0102
Subject(s) - placebo , medicine , cerebral palsy , clinical endpoint , anesthesia , randomized controlled trial , gross motor function classification system , physical therapy , alternative medicine , pathology
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 × 10 7 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 × 10 7 /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. S tem C ells T ranslational M edicine 2017;6:2071–2078

The content you want is available to Zendy users.

Already have an account? Click here to sign in.
Having issues? You can contact us here