Haploidentical donor transplants for severe aplastic anemia

Abstract

Haploidentical stem cell transplantation (HAPLO) is being increasingly used, and significant progress has been made both with ex vivo T-cell depleted grafts as well as with unmanipulated marrow or peripheral blood grafts. We here review the current status of HAPLO grafts in patients with acquired severe aplastic anemia. Several transplant platforms have been tested, to overcome graft severe rejection and graft vs host disease (GvHD): these include differences in the conditioning regimen, in graft source and graft manipulation, and in GvHD prophylaxis. The latter include ex vivo T-cell depletion and/or antithymocyte globulin and/or high dose post-transplant cyclophosphamide. Some programs also include the use of marrow or cord blood mesenchymal stem cells, infused at the time of transplantation. Extremely encouraging results are being reported especially in the pediatric population, but also in young adults: we will review reports on 375 patients, with an average rejection rate of 6%, grade II-IV GvHD of 23% and 1-year survival of 80%. Finally we will discuss the place of HAPLO transplants in the context of alternative donor grafts for acquired aplastic anemia.