HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders
Alice Bertaina, Pietro Merli, Sergio Rutella, Daria Pagliara, Maria Ester Bernardo, Riccardo Masetti, Daniela Pende, Michela Falco, Rupert Handgretinger, Francesca Moretta, Barbarella Lucarelli, Letizia P Brescia, Giuseppina Li Pira, Manuela Testi, Caterina Cancrini, Nabil Kabbara, Rita Carsetti, Andrea Finocchi, Alessandro Moretta, Lorenzo Moretta, Franco Locatelli, Alice Bertaina, Pietro Merli, Sergio Rutella, Daria Pagliara, Maria Ester Bernardo, Riccardo Masetti, Daniela Pende, Michela Falco, Rupert Handgretinger, Francesca Moretta, Barbarella Lucarelli, Letizia P Brescia, Giuseppina Li Pira, Manuela Testi, Caterina Cancrini, Nabil Kabbara, Rita Carsetti, Andrea Finocchi, Alessandro Moretta, Lorenzo Moretta, Franco Locatelli
Abstract
Twenty-three children with nonmalignant disorders received HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) after ex vivo elimination of αβ(+) T cells and CD19(+) B cells. The median number of CD34(+), αβ(+)CD3(+), and B cells infused was 16.8 × 10(6), 40 × 10(3), and 40 × 10(3) cells/kg, respectively. No patient received any posttransplantation pharmacologic prophylaxis for graft-versus-host disease (GVHD). All but 4 patients engrafted, these latter being rescued by a second allograft. Three patients experienced skin-only grade 1 to 2 acute GVHD. No patient developed visceral acute or chronic GVHD. Cumulative incidence of transplantation-related mortality was 9.3%. With a median follow-up of 18 months, 21 of 23 children are alive and disease-free, the 2-year probability of disease-free survival being 91.1%. Recovery of γδ(+) T cells was prompt, but αβ(+) T cells progressively ensued over time. Our data suggest that this novel graft manipulation strategy is safe and effective for haplo-HSCT. This trial was registered at www.clinicaltrials.gov as #NCT01810120.
© 2014 by The American Society of Hematology.
Source: PubMed