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

Giulia Ceglie; Laura Papetti; Lorenzo Figà Talamanca; Barbarella Lucarelli; Mattia Algeri; Stefania Gaspari; Giuseppina Li Pira; Giovanna-Stefania Colafati; Mauro Montanari; Massimiliano Valeriani; Franco Locatelli; Pietro Merli

doi:10.1002/acn3.50843

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

Giulia Ceglie, Laura Papetti, Lorenzo Figà Talamanca, Barbarella Lucarelli, Mattia Algeri, Stefania Gaspari, Giuseppina Li Pira, Giovanna-Stefania Colafati, Mauro Montanari, Massimiliano Valeriani, Franco Locatelli, Pietro Merli

Ospedale Pediatrico Bambino Gesu

Research output: Contribution to journal › Article › peer-review

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	https://doi.org/10.1002/acn3.50843
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 › peer-review

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. In: Annals of Clinical and Translational Neurology. 2019.

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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.",

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AU - Papetti, Laura

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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

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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.

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JO - Annals of Clinical and Translational Neurology

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