Haploidentical transplantation in patients with acquired aplastic anemia

Haploidentical SCT (haplo-SCT) has been considered a therapeutic option in patients with acquired severe aplastic anemia (SAA) failing at least one course of immune suppressive therapy with antithymocyte globulin and lacking an HLA-matched related or unrelated donor. The platforms of both ex vivo T-cell-depleted and unmanipulated grafts have been explored in children and adults. Overall, the primary objective of a stable haploidentical hematopoietic engraftment with a low rate of GVHD is unmet in a significant proportion of patients undergoing haplo-SCT for SAA. Haploidentical transplants for refractory SAA should be performed in a specialist center with major experience in hematopoietic SCT procedures and preferably performed within the framework of a local clinical protocol designed specifically to address the prevention of graft rejection and GVHD.