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T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica
Neuromyelitis optica is an immune‐mediated disease characterized by a relapsing course, resulting in progressive disability. In children, given the long life expectancy, a disease‐modifying treatment could be particularly desirable. Unfortunately, the currently available treatment strategies with th...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6801170/ https://www.ncbi.nlm.nih.gov/pubmed/31529689 http://dx.doi.org/10.1002/acn3.50843 |
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author | Ceglie, Giulia Papetti, Laura Figà Talamanca, Lorenzo Lucarelli, Barbarella Algeri, Mattia Gaspari, Stefania Li Pira, Giuseppina Colafati, Giovanna‐Stefania Montanari, Mauro Valeriani, Massimiliano Locatelli, Franco Merli, Pietro |
author_facet | Ceglie, Giulia Papetti, Laura Figà Talamanca, Lorenzo Lucarelli, Barbarella Algeri, Mattia Gaspari, Stefania Li Pira, Giuseppina Colafati, Giovanna‐Stefania Montanari, Mauro Valeriani, Massimiliano Locatelli, Franco Merli, Pietro |
author_sort | Ceglie, Giulia |
collection | PubMed |
description | Neuromyelitis optica is an immune‐mediated disease characterized by a relapsing course, resulting in progressive disability. In children, given the long life expectancy, a disease‐modifying treatment could be particularly desirable. Unfortunately, the currently available treatment strategies with this potential are scarce. Very limited data are available about the use of allogeneic hematopoietic stem cell transplantation (HSCT) for autoimmune neurological diseases. In this report, we present a pediatric case successfully treated with allogeneic HSCT from an HLA‐haploidentical donor, after ex vivo TCR/CD19‐depletion of the graft. To the best of our knowledge, this is the first case of a pediatric patient to benefit from such a treatment. |
format | Online Article Text |
id | pubmed-6801170 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-68011702019-10-22 T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica Ceglie, Giulia Papetti, Laura Figà Talamanca, Lorenzo Lucarelli, Barbarella Algeri, Mattia Gaspari, Stefania Li Pira, Giuseppina Colafati, Giovanna‐Stefania Montanari, Mauro Valeriani, Massimiliano Locatelli, Franco Merli, Pietro Ann Clin Transl Neurol Brief Communications Neuromyelitis optica is an immune‐mediated disease characterized by a relapsing course, resulting in progressive disability. In children, given the long life expectancy, a disease‐modifying treatment could be particularly desirable. Unfortunately, the currently available treatment strategies with this potential are scarce. Very limited data are available about the use of allogeneic hematopoietic stem cell transplantation (HSCT) for autoimmune neurological diseases. In this report, we present a pediatric case successfully treated with allogeneic HSCT from an HLA‐haploidentical donor, after ex vivo TCR/CD19‐depletion of the graft. To the best of our knowledge, this is the first case of a pediatric patient to benefit from such a treatment. John Wiley and Sons Inc. 2019-09-17 /pmc/articles/PMC6801170/ /pubmed/31529689 http://dx.doi.org/10.1002/acn3.50843 Text en © 2019 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association. This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Brief Communications Ceglie, Giulia Papetti, Laura Figà Talamanca, Lorenzo Lucarelli, Barbarella Algeri, Mattia Gaspari, Stefania Li Pira, Giuseppina Colafati, Giovanna‐Stefania Montanari, Mauro Valeriani, Massimiliano Locatelli, Franco Merli, Pietro T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title | T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title_full | T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title_fullStr | T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title_full_unstemmed | T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title_short | T‐cell depleted HLA‐haploidentical HSCT in a child with neuromyelitis optica |
title_sort | t‐cell depleted hla‐haploidentical hsct in a child with neuromyelitis optica |
topic | Brief Communications |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6801170/ https://www.ncbi.nlm.nih.gov/pubmed/31529689 http://dx.doi.org/10.1002/acn3.50843 |
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