Therapeutic outcomes of haploidentical allogeneic hematopoietic stem cell transplantation in patients with severe aplastic anemia: a multicenter study

Background Haploidentical donor allogeneic hematopoietic stem cell transplantation (HID-HSCT) is an alternative curative treatment for patients with severe aplastic anemia (SAA) who do not have suitable matched related donors (MRD). The aim of this study was to compare the therapeutic outcomes of HID-HSCT with those of MRD-HSCT for SAA. Methods A total of 235 SAA patients who underwent HID-HSCT (116) or MRD-HSCT (119) at 11 transplantation centers from January 2007 to January 2016 were included. Complications and survival outcomes were evaluated and compared between the 2 groups. Results The HID group had a lower incidence of secondary graft failure (GF) but higher incidences of acute graft-versus-host disease (aGVHD) and chronic GVHD (cGVHD). However, the incidence of severe aGVHD (grade III-IV), poor graft function (PGF), and infections was comparable between groups. Patients in the HID group had a significantly lower survival and overall survival rates than those in the MRD group. The estimated 3-year survival rates for the MRD and HID groups were 82.82% and 75.00%, respectively. Ferritin levels, GF, PGF, severe aGVHD, and infections were the significant risk factors for survival. Conclusion The overall survival rate is acceptable for patients who underwent HID-HSCT, making it a feasible treatment choice for SAA patients. Corresponding author: Professor Yang Xiao, Stem Cell Translational Medicine Center, The Second Affiliated Hospital, Guangzhou Medical University, Guangzhou, China & Guangzhou General Hospital of Guangzhou Military Command, Guangzhou, China. Email: jdxiao111@163.com Yonghua Li, Fengqi Duan and Haowen Xiao contributed equally to this work. Author contributions Yonghua Li and Yang Xiao designed the research. Yonghua Li and Fengqi Duan analyzed the data and wrote the manuscript. All authors conducted the research, collected the clinical data, and approved the submitted and final versions of the manuscript. Conflicts of interest The authors declare that there are no conflicts of interest. Funding This work was partly supported by the State Natural Sciences Fund (Project number 81570107), Natural Science Foundation of Guangdong Province (Project number 2014A030311006) and Guangzhou Pearl River Scientific and Technological New Star Capitals (grant no. 2012 J2200008). Copyright © 2018 Wolters Kluwer Health, Inc. All rights reserved.

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