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.

T-cell depleted HLA-haploidentical HSCT in a child with neuromyelitis optica / Ceglie, G.; Papetti, L.; Figa Talamanca, L.; Lucarelli, B.; Algeri, M.; Gaspari, S.; Li Pira, G.; Colafati, G. -S.; Montanari, M.; Valeriani, M.; Locatelli, F.; Merli, P.. - In: ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY. - ISSN 2328-9503. - 6:10(2019), pp. 2110-2113. [10.1002/acn3.50843]

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

Locatelli F.;
2019

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.
2019
neuromyelitis optica; child; T-cell
01 Pubblicazione su rivista::01i Case report
T-cell depleted HLA-haploidentical HSCT in a child with neuromyelitis optica / Ceglie, G.; Papetti, L.; Figa Talamanca, L.; Lucarelli, B.; Algeri, M.; Gaspari, S.; Li Pira, G.; Colafati, G. -S.; Montanari, M.; Valeriani, M.; Locatelli, F.; Merli, P.. - In: ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY. - ISSN 2328-9503. - 6:10(2019), pp. 2110-2113. [10.1002/acn3.50843]
File allegati a questo prodotto
File Dimensione Formato  
Ceglie_T-cell_2019.pdf

accesso aperto

Tipologia: Versione editoriale (versione pubblicata con il layout dell'editore)
Licenza: Creative commons
Dimensione 355.06 kB
Formato Adobe PDF
355.06 kB Adobe PDF

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1481705
Citazioni
  • ???jsp.display-item.citation.pmc??? 4
  • Scopus 13
  • ???jsp.display-item.citation.isi??? 11
social impact