Twenty-three children with nonmalignant disorders received HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) after ex vivo elimination of alpha beta(+) T cells and CD19(+) B cells. The median number of CD34(+), alpha beta(+)CD3(+), and B cells infusedwas 16.8 x 10(6), 40 x 10(3), and 40 x 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 gamma delta(+) T cells was prompt, but alpha beta(+) T cells progressively ensued over time. Our datasuggest that thisnovelgraftmanipulation strategy is safe and effective for haplo-HSCT. This trial was registered at www.clinicaltrials.gov as #NCT01810120.

Bertaina, A., Merli, P., Rutella, S., Pagliara, D., Bernardo, M. E., Masetti, R., Pende, D., Falco, M., Handgretinger, R., Moretta, F., Lucarelli, B., Brescia, L. P., Li Pira, G., Testi, M., Cancrini, C., Kabbara, N., Carsetti, R., Finocchi, A., Moretta, A., Moretta, L., Locatelli, F., HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders, <<BLOOD>>, 2014; 124 (5): 822-826. [doi:10.1182/blood-2014-03-563817] [https://hdl.handle.net/10807/242415]

HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders

Masetti, Riccardo;Carsetti, Rita;Locatelli, Franco
2014

Abstract

Twenty-three children with nonmalignant disorders received HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) after ex vivo elimination of alpha beta(+) T cells and CD19(+) B cells. The median number of CD34(+), alpha beta(+)CD3(+), and B cells infusedwas 16.8 x 10(6), 40 x 10(3), and 40 x 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 gamma delta(+) T cells was prompt, but alpha beta(+) T cells progressively ensued over time. Our datasuggest that thisnovelgraftmanipulation strategy is safe and effective for haplo-HSCT. This trial was registered at www.clinicaltrials.gov as #NCT01810120.
2014
Inglese
Bertaina, A., Merli, P., Rutella, S., Pagliara, D., Bernardo, M. E., Masetti, R., Pende, D., Falco, M., Handgretinger, R., Moretta, F., Lucarelli, B., Brescia, L. P., Li Pira, G., Testi, M., Cancrini, C., Kabbara, N., Carsetti, R., Finocchi, A., Moretta, A., Moretta, L., Locatelli, F., HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders, <<BLOOD>>, 2014; 124 (5): 822-826. [doi:10.1182/blood-2014-03-563817] [https://hdl.handle.net/10807/242415]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/242415
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