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
2019 Annals of Clinical and Translational Neurology  
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
more » ... ediatric 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.
doi:10.1002/acn3.50843 pmid:31529689 pmcid:PMC6801170 fatcat:b5wfdr7ztvhu3aqfj5bpr65j7i