Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial.
| dc.contributor.author | Sun, Jessica M | |
| dc.contributor.author | Case, Laura E | |
| dc.contributor.author | McLaughlin, Colleen | |
| dc.contributor.author | Burgess, Alicia | |
| dc.contributor.author | Skergan, Natalie | |
| dc.contributor.author | Crane, Sydney | |
| dc.contributor.author | Jasien, Joan M | |
| dc.contributor.author | Mikati, Mohamad A | |
| dc.contributor.author | Troy, Jesse | |
| dc.contributor.author | Kurtzberg, Joanne | |
| dc.date.accessioned | 2022-09-13T15:49:25Z | |
| dc.date.available | 2022-09-13T15:49:25Z | |
| dc.date.issued | 2022-07-10 | |
| dc.date.updated | 2022-09-13T15:49:25Z | |
| dc.description.abstract | AimTo evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy (CP).MethodNinety-one children (52 males, 39 females; median age 3 years 7 months [range 2-5 years]) with CP due to hypoxic-ischemic encephalopathy, stroke, or periventricular leukomalacia were randomized to three arms: (1) the AlloCB group received 10 × 107 AlloCB total nucleated cells (TNC) per kilogram at baseline (n = 31); (2) the hCT-MSC group received 2 × 106 hCT-MSC at baseline, 3 months, and 6 months (n = 28); (3) the natural history control group received 10 × 107 AlloCB TNC per kilogram at 12 months (n = 31). Motor function was assessed with the Gross Motor Function Measure-66 (GMFM-66) and Peabody Developmental Motor Scale, Second Edition.ResultsInfusions (n = 143) were well tolerated, with eight infusion reactions (three in the AlloCB group, five in hCT-MSC) and no other safety concerns. At 12 months, the mean differences (95% confidence intervals [CI]) between actual and expected changes in GMFM-66 score were AlloCB 5.8 points (3.4-8.2), hCT-MSC 4.3 (2.2-6.4), and natural history 3.1 (1.4-5.0). In exploratory, post hoc analysis, the mean GMFM-66 score (95% CI) of the hCT-MSC group was 1.4 points higher than natural history (-1.1 to 4.0; p = 0.27), and the AlloCB group was 3.3 points higher than natural history (0.59-5.93; p = 0.02) after adjustment for baseline Gross Motor Function Classification System level, GMFM-66 score, and etiology.InterpretationHigh-dose AlloCB is a potential cell therapy for CP and should be further tested in a randomized, blinded, placebo-controlled trial. | |
| dc.identifier.issn | 0012-1622 | |
| dc.identifier.issn | 1469-8749 | |
| dc.identifier.uri | ||
| dc.language | eng | |
| dc.publisher | Wiley | |
| dc.relation.ispartof | Developmental medicine and child neurology | |
| dc.relation.isversionof | 10.1111/dmcn.15325 | |
| dc.title | Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial. | |
| dc.type | Journal article | |
| duke.contributor.orcid | Sun, Jessica M|0000-0001-8085-1013 | |
| duke.contributor.orcid | Case, Laura E|0000-0002-2941-2186 | |
| duke.contributor.orcid | Mikati, Mohamad A|0000-0003-0363-8715 | |
| duke.contributor.orcid | Troy, Jesse|0000-0001-5410-8146 | |
| duke.contributor.orcid | Kurtzberg, Joanne|0000-0002-3370-0703 | |
| pubs.organisational-group | Duke | |
| pubs.organisational-group | School of Medicine | |
| pubs.organisational-group | Basic Science Departments | |
| pubs.organisational-group | Clinical Science Departments | |
| pubs.organisational-group | Institutes and Centers | |
| pubs.organisational-group | Biostatistics & Bioinformatics | |
| pubs.organisational-group | Neurobiology | |
| pubs.organisational-group | Orthopaedic Surgery | |
| pubs.organisational-group | Pathology | |
| pubs.organisational-group | Pediatrics | |
| pubs.organisational-group | Pediatrics, Hematology-Oncology | |
| pubs.organisational-group | Pediatrics, Medical Genetics | |
| pubs.organisational-group | Pediatrics, Neurology | |
| pubs.organisational-group | Duke Cancer Institute | |
| pubs.organisational-group | Institutes and Provost's Academic Units | |
| pubs.organisational-group | University Institutes and Centers | |
| pubs.organisational-group | Duke Institute for Brain Sciences | |
| pubs.organisational-group | Duke-UNC Center for Brain Imaging and Analysis | |
| pubs.organisational-group | Initiatives | |
| pubs.organisational-group | Duke Science & Society | |
| pubs.organisational-group | Orthopaedic Surgery, Physical Therapy | |
| pubs.organisational-group | Duke Innovation & Entrepreneurship | |
| pubs.organisational-group | Pediatrics, Transplant and Cellular Therapy | |
| pubs.organisational-group | Regeneration Next Initiative | |
| pubs.publication-status | Published |