Current insights into the treatments of severe aplastic anemia in China.

Abstract

Recently, several studies have been conducted to generate considerable evidence regarding unique treatments for severe aplastic anemia (SAA) in China. Haploidentical donor hematopoietic stem cell transplantation (HID-HSCT) showed an overall survival rate (80.3-86.1%) comparable to those with immunosuppressive therapy (IST) and matched related donor (MRD)- and matched unrelated donor (MUD)-HSCT. Failure-free survival of HID-HSCT was also comparable (76.4-85.0%) to those of MRD- and MUD-HSCT and better than IST in patients < 40years. Although these results are promising, HID-HSCT should be regarded as a salvage therapy when young patients fail to respond to IST. Porcine anti-human lymphocyte immunoglobulin (pALG) showed similar or superior overall response at 6months compared to rabbit anti-human thymocyte immunoglobulin (rATG) (64.0-79.4% in the pALG-group vs.48.1-64.7% in the rATG-group) as a first-line IST. Promising hematological response (28.4-33.3%) was observed in patients with refractory AA following infusion of the mesenchymal stromal cells (MSCs) derived from the bone marrow of allogeneic donors. pALG can replace rATG as an immunosuppressive drug and MSCs infusion can be used as a second-line treatment for refractory SAA. We believe that this review contributes to refine the global practices for SAA treatment.