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 infusedwas 16.8 × 106, 40 × 103, and 40 × 103 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 datasuggest that thisnovelgraftmanipulation 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.
HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders / 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.; Pira, G. L.; Testi, M.; Cancrini, C.; Kabbara, N.; Carsetti, R.; Finocchi, A.; Moretta, A.; Moretta, L.; Locatelli, F.. - In: BLOOD. - ISSN 0006-4971. - 124:5(2014), pp. 822-826. [10.1182/blood-2014-03-563817]
HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders
Locatelli F.
2014
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 infusedwas 16.8 × 106, 40 × 103, and 40 × 103 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 datasuggest that thisnovelgraftmanipulation 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
