Removal of αβ+ T and CD19+ B cells is an effective strategy for successful HLA-haploidentical hematopoietic stem cell transplantation.
The high probability of disease-free survival renders this transplant option attractive for any child with non-malignant disorders.
Twenty-three children (15 males, 8 females, median age 3.3 years) with non-malignant 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 16.8x106/kg, 40x103/kg and 40x103/kg, respectively. No patient received any post-transplantation pharmacological graft-versus-host disease (GvHD) prophylaxis. All patients but 4 engrafted, these latter being rescued by a second allograft. Three patients experienced skin-only grade I-II 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 (range 5-40), 21/23 children are alive and disease-free, the 2-year probability of disease-free survival being 91.1%. Recovery of γδ+ T cells was prompt, while αβ+ T cells progressively ensued over time. Our data suggest that this novel graft manipulation strategy is safe and effective for HLA-haplo-HSCT. This study was registered at clinicaltrials.gov, identifier: NCT01810120.
- Submitted March 21, 2014.
- Accepted May 16, 2014.
- Copyright © 2014 American Society of Hematology