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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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Detalles Bibliográficos
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
Descripción
Sumario: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.