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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...

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Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
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.
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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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