Haploidentical stem cell transplantation for aplastic anemia: the current advances and future challenges

Abstract

Haematopoietic stem cell transplantation (HSCT) is a curative option for severe aplastic anemia (SAA). Finding a suitable matched donor in a timely manner is a challenge. The availability of haploidentical donors and their successful use in transplantation have expanded valid choices for SAA. In recent decades, haploidentical HSCT (haplo-HSCT) for the treatment of SAA has been continuously attempted, and great strides have been made. Nowadays, haplo-HSCT using different regimens has overcome the difficulty of graft failure and severe graft-versus-host disease (GvHD), and achieved inspiring survival outcomes in SAA. The regimens consist mainly of granulocyte colony-stimulating factor (G-CSF) plus antithymocyte globulin (ATG), posttransplantation cyclophosphamide (PT-Cy), and ex vivo graft T-cell depletion (TCD). In particular, the G-CSF and ATG-based regimen includes the largest sample size and the successful wide use of the G-CSF and ATG-based regimen has promoted haplo-HSCT a higher priority in SAA patients without matched related or unrelated donors in China. Recent studies have also indicated that haplo-HSCT using PT-Cy or TCD regimen is a practicable alternative, but the sample size is relatively small. Here, we offer an overview of clinical results obtained through the use of haploidentical transplantation in SAA, mainly focusing on current advances and future challenges.