Progress in Haploidentical Stem Cell Transplantation

Haploidentical stem cell transplantation is an attractive form of transplantation because of the immediate donor availability, ease of stem cell procurement, and the possibility to further collect donor cells for cellular therapy. Historically, maintaining T cells in the graft has been associated with very high rates of graft-versus-host-disease (GVHD), whereas T cell–depleted haploidentical transplantation has been limited by a higher incidence of graft rejection and nonrelapse mortality related to infectious complications as a result of delayed immune reconstitution posttransplantation. Recent approaches have attempted to eliminate the alloreactive T cells to prevent GVHD posttransplantation. Administration of high-dose cyclophosphamide early posttransplantation in combination with tacrolimus and mycophenolate mofetil has produced engraftment and GVHD rates similar to HLA-matched sibling transplants, suggesting that the most important barriers against successful haploidentical transplantation can be overcome. Future directions should focus on optimizing conditioning regimens for different diseases and prevention of disease relapse posttransplantation.