Infusion of BPX-501 T Cells Renders Haplo-HSCT a First-Line Option for Children with PI

A Phase I/II study indicates that haplo identical hematopoietic stem cell transplantation (haplo-HSCT), after depletion of α/β T cells and B cells followed by adoptive infusion of donorBPX-501 cells, is an effective alternative for children with primary immunodeficiency disease (PI) in need of an urgent allograft or lacking a suitable human leukocyte antigen (HLA)-matched donor.