Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease.
Brain
/ diagnostic imaging
Child, Preschool
Follow-Up Studies
HLA Antigens
/ immunology
Humans
Infant
Isoantibodies
/ blood
Magnetic Resonance Imaging
Male
Neural Stem Cells
/ cytology
Pelizaeus-Merzbacher Disease
/ immunology
Severity of Illness Index
Stem Cell Transplantation
/ adverse effects
Transplantation, Homologous
Treatment Outcome
Pelizaeus-Merzbacher disease
leukodystrophy
myelin
neural stem cells
neurodegenerative disease
oligodendrocyte
pediatric
transplantation
Journal
Stem cell reports
ISSN: 2213-6711
Titre abrégé: Stem Cell Reports
Pays: United States
ID NLM: 101611300
Informations de publication
Date de publication:
13 08 2019
13 08 2019
Historique:
received:
12
11
2018
revised:
04
07
2019
accepted:
05
07
2019
pubmed:
6
8
2019
medline:
24
4
2020
entrez:
6
8
2019
Statut:
ppublish
Résumé
Four boys with Pelizaeus-Merzbacher disease, an X-linked leukodystrophy, underwent transplantation with human allogeneic central nervous system stem cells (HuCNS-SC). Subsequently, all subjects were followed for an additional 4 years in this separate follow-up study to evaluate safety, neurologic function, magnetic resonance imaging (MRI) data, and immunologic response. The neurosurgical procedure, immunosuppression, and HuCNS-SC transplantation were well tolerated and all four subjects were alive at the conclusion of the study period. At year 2, all subjects exhibited diffusion MRI changes at the implantation sites as well as in more distant brain regions. There were persistent, increased signal changes in the three patients who were studied up to year 5. Two of four subjects developed donor-specific HLA alloantibodies, demonstrating that neural stem cells can elicit an immune response when injected into the CNS, and suggesting the importance of monitoring immunologic parameters and identifying markers of engraftment in future studies.
Identifiants
pubmed: 31378671
pii: S2213-6711(19)30254-1
doi: 10.1016/j.stemcr.2019.07.002
pmc: PMC6700500
pii:
doi:
Substances chimiques
HLA Antigens
0
Isoantibodies
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
254-261Subventions
Organisme : Medical Research Council
ID : MC_PC_12009
Pays : United Kingdom
Informations de copyright
Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.
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