T-cell depleted HLA-haploidentical HSCT in a child with neuromyelitis optica

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Abstract

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.

Original languageEnglish
JournalAnnals of Clinical and Translational Neurology
DOIs
Publication statusE-pub ahead of print - Sep 17 2019

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Neuromyelitis Optica
Hematopoietic Stem Cell Transplantation
T-Lymphocytes
Pediatrics
Immune System Diseases
Life Expectancy
Autoimmune Diseases
Therapeutics
Tissue Donors
Transplants

Cite this

@article{108b8c81f7ea46f7a235700dd9fc932b,
title = "T-cell depleted HLA-haploidentical HSCT in a child with neuromyelitis optica",
abstract = "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.",
author = "Giulia Ceglie and Laura Papetti and {Fig{\`a} Talamanca}, Lorenzo and Barbarella Lucarelli and Mattia Algeri and Stefania Gaspari and {Li Pira}, Giuseppina and Giovanna-Stefania Colafati and Mauro Montanari and Massimiliano Valeriani and Franco Locatelli and Pietro Merli",
note = "{\circledC} 2019 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association.",
year = "2019",
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doi = "10.1002/acn3.50843",
language = "English",
journal = "Annals of Clinical and Translational Neurology",
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TY - JOUR

T1 - T-cell depleted HLA-haploidentical HSCT in a child with neuromyelitis optica

AU - Ceglie, Giulia

AU - Papetti, Laura

AU - Figà Talamanca, Lorenzo

AU - Lucarelli, Barbarella

AU - Algeri, Mattia

AU - Gaspari, Stefania

AU - Li Pira, Giuseppina

AU - Colafati, Giovanna-Stefania

AU - Montanari, Mauro

AU - Valeriani, Massimiliano

AU - Locatelli, Franco

AU - Merli, Pietro

N1 - © 2019 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association.

PY - 2019/9/17

Y1 - 2019/9/17

N2 - 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.

AB - 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.

U2 - 10.1002/acn3.50843

DO - 10.1002/acn3.50843

M3 - Article

JO - Annals of Clinical and Translational Neurology

JF - Annals of Clinical and Translational Neurology

SN - 2328-9503

ER -