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 language | English |
|---|---|
| Journal | Annals of Clinical and Translational Neurology |
| DOIs | |
| Publication status | E-pub ahead of print - Sep 17 2019 |
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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.
In: Annals of Clinical and Translational Neurology, 17.09.2019.Research output: Contribution to journal › Article
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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 -