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Sibling umbilical cord blood infusion is safe in young children with cerebral palsy.

Publication ,  Journal Article
Sun, JM; Case, LE; Mikati, MA; M Jasien, J; McLaughlin, C; Waters-Pick, B; Worley, G; Troy, J; Kurtzberg, J
Published in: Stem Cells Transl Med
September 2021

Preclinical and early phase clinical studies suggest that an appropriately dosed umbilical cord blood (CB) infusion has the potential to help improve motor function in young children with cerebral palsy (CP). As many children with CP do not have their own CB available, use of allogeneic cells would extend access to this potentially beneficial therapy to more children. In this phase I, open-label study, 15 children, aged 1 to 6 years, with moderate to severe spastic CP were treated with a single intravenous infusion of allogeneic human leukocyte antigen (HLA) matched or partially matched sibling CB with a cell dose of ≥2.5 × 107 cells/kg based on the pre-cryopreservation count (median infused cell dose, 3.3 × 107 ; range, 1.8-5.2 × 107 ). There were a total of 49 adverse events (AEs) over a 2-year time period, but there were no AEs related to the CB infusions. Specifically, there were no acute infusion reactions and no antibody formation against platelets, red blood cells, or donor-specific HLA antigens. Donor cells were not detected in peripheral blood 6 months later. Six months after infusion, participants were assessed for response and experienced a mean ± SD increase of 4.7 ± 2.5 points on the Gross Motor Function Measure-66 and 1 ± 2.9 points on the Peabody Gross Motor Quotient. Appropriately dosed, allogeneic partially or fully HLA-matched sibling CB infusion is well tolerated and potentially beneficial in young children with CP.

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Published In

Stem Cells Transl Med

DOI

EISSN

2157-6580

Publication Date

September 2021

Volume

10

Issue

9

Start / End Page

1258 / 1265

Location

England

Related Subject Headings

  • Siblings
  • Infant
  • Humans
  • HLA Antigens
  • Graft vs Host Disease
  • Fetal Blood
  • Child, Preschool
  • Child
  • Cerebral Palsy
  • 4003 Biomedical engineering
 

Citation

APA
Chicago
ICMJE
MLA
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Sun, J. M., Case, L. E., Mikati, M. A., M Jasien, J., McLaughlin, C., Waters-Pick, B., … Kurtzberg, J. (2021). Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. Stem Cells Transl Med, 10(9), 1258–1265. https://doi.org/10.1002/sctm.20-0470
Sun, Jessica M., Laura E. Case, Mohamad A. Mikati, Joan M Jasien, Colleen McLaughlin, Barbara Waters-Pick, Gordon Worley, Jesse Troy, and Joanne Kurtzberg. “Sibling umbilical cord blood infusion is safe in young children with cerebral palsy.Stem Cells Transl Med 10, no. 9 (September 2021): 1258–65. https://doi.org/10.1002/sctm.20-0470.
Sun JM, Case LE, Mikati MA, M Jasien J, McLaughlin C, Waters-Pick B, et al. Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. Stem Cells Transl Med. 2021 Sep;10(9):1258–65.
Sun, Jessica M., et al. “Sibling umbilical cord blood infusion is safe in young children with cerebral palsy.Stem Cells Transl Med, vol. 10, no. 9, Sept. 2021, pp. 1258–65. Pubmed, doi:10.1002/sctm.20-0470.
Sun JM, Case LE, Mikati MA, M Jasien J, McLaughlin C, Waters-Pick B, Worley G, Troy J, Kurtzberg J. Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. Stem Cells Transl Med. 2021 Sep;10(9):1258–1265.

Published In

Stem Cells Transl Med

DOI

EISSN

2157-6580

Publication Date

September 2021

Volume

10

Issue

9

Start / End Page

1258 / 1265

Location

England

Related Subject Headings

  • Siblings
  • Infant
  • Humans
  • HLA Antigens
  • Graft vs Host Disease
  • Fetal Blood
  • Child, Preschool
  • Child
  • Cerebral Palsy
  • 4003 Biomedical engineering