Haploidentical haematopoietic stem cell transplantation for the treatment of severe aplastic anaemia patients with high-risk factors who lack an HLA-matched sibling donor.

This study aimed to analyse the efficacy of haploidentical donor (HID) haematopoietic stem cell transplantation as a first-line treatment for severe aplastic anaemia (SAA) with high-risk factors (infection or very severe aplastic anaemia,VSAA) in patients who lack an HLA-matched sibling donor (MSD). The patients with infection were treated with anti-infection therapy, and allogeneic haematopoietic stem cell transplantation (HSCT) was carried out after the infection being effectively controlled was in accordance with the stable infection (SI) standard. A total of 44 SAA patients receiving MSD transplantation (n=19) and HID transplantation (n=25) were included in this study. There was no significant difference in neutrophil engraftment between the two groups [MSD vs. HID, 19 (11-38) vs. 22 (15-47).P=0.241], and the difference in platelet engraftment was statistically significant [MSD vs. HID, 11(7-33) vs. 20 (12-69), P=0.034]. The HID group exhibited a higher incidence of grade II-IV acute graft-versus-host disease (aGVHD) (HID vs. MSD, 48.0% vs10.5%, P=0.034)and a higher incidence of chronic GVHD (cGVHD) than the MSD group (64.0% vs. 21.1%, P=0.026). There was no significant difference between overall survival (OS) following HID and MSD transplantation (84.0% vs. 89.5%, P=0.664) and failure-free survival (FFS)(80.0% vs. 84.2%, P=0.965). The interval from diagnosis to transplantation (>50d) and ECOG (>2) were independent factors associated with OS and FFS. HID HSCT may be an effective and safe option for SAA patients with high-risk factors who lack an MSD.