title: Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant.
abstract: BACKGROUND
  Haploidentical donor (HID) hematopoietic stem cell transplantation (HSCT) is an alternative treatment method for severe aplastic anemia (SAA) patients lacking suitable identical donors and those who are refractory to immunosuppressive therapy (IST). The current study evaluated the feasibility of upfront haploidentical HSCT in SAA patients.
  
  
  METHODS
  We conducted a multicenter study based on a registry database. One hundred fifty-eight SAA patients who underwent upfront transplantation between June 2012 and September 2015 were enrolled.
  
  
  RESULTS
  Eighty-nine patients had haploidentical donors (HIDs), and 69 had matched related donors (MRDs) for HSCT. The median times for myeloid engraftment in the HID and MRD cohorts were 12 (range, 9-20) and 11 (range, 8-19) days, with a cumulative incidence of 97.8 and 97.1% (P = 0.528), respectively. HID recipients had an increased cumulative incidence of grades II-IV acute graft-versus-host disease (aGVHD) (30.3 vs. 1.5%, P < 0.001), grades III-IV aGVHD (10.1 vs. 1.5%, P = 0.026), and chronic GVHD (cGVHD) (30.6 vs. 4.4%, P < 0.001) at 1 year but similar extensive cGVHD (3.4 vs. 0%, P = 0.426). The three-year estimated overall survival (OS) rates were 86.1 and 91.3% (P = 0.358), while the three-year estimated failure-free survival (FFS) rates were 85.0 and 89.8% (P = 0.413) in the HID and MRD cohorts, respectively. In multivariate analysis, survival outcome for the entire population was significantly adversely associated with increased transfusions and poor performance status pre-SCT. We did not observe differences in primary engraftment and survival outcomes by donor type.
  
  
  CONCLUSIONS
  Haploidentical SCT as upfront therapy was an effective and safe option for SAA patients, with favorable outcomes in experienced centers.
authors.affiliation: Peking University Institute of Hematology, Peking University People's Hospital, No. 11 Xizhimen South Street, Xicheng District, Beijing, 100044, People's Republic of China.
authors.forename: Lan-Ping
authors.initials: LP
authors.lastname: Xu
authors.affiliation: The First Affiliated Hospital of Soochow University, Soochow, China.
authors.forename: Song
authors.initials: S
authors.lastname: Jin
authors.affiliation: Guangzhou First People's Hospital, Guangzhou, China.
authors.forename: Shun-Qing
authors.initials: SQ
authors.lastname: Wang
authors.affiliation: Xiehe Hospital affiliated to Huazhong University of Science and Technology, Wuhan, China.
authors.forename: Ling-Hui
authors.initials: LH
authors.lastname: Xia
authors.affiliation: Lanzhou Military Area General Hospital, Lanzhou, China.
authors.forename: Hai
authors.initials: H
authors.lastname: Bai
authors.affiliation: The First Hospital of Jilin University, Changchun, China.
authors.forename: Su-Jun
authors.initials: SJ
authors.lastname: Gao
authors.affiliation: Nanfang Hospital Affiliated to Southern Medical University, Guangzhou, China.
authors.forename: Qi-Fa
authors.initials: QF
authors.lastname: Liu
authors.affiliation: Changhai Hospital affiliated to Second Military Medical University, Shanghai, China.
authors.forename: Jian-Min
authors.initials: JM
authors.lastname: Wang
authors.affiliation: Shandong Provincial Hospital, Jinan, China.
authors.forename: Xin
authors.initials: X
authors.lastname: Wang
authors.affiliation: The First Affiliated Hospital of Xinjiang Medical University, Urumchi, China.
authors.forename: Ming
authors.initials: M
authors.lastname: Jiang
authors.affiliation: Xinqiao Hospital Affiliated to Third Military Medical University, Chongqing, China.
authors.forename: Xi
authors.initials: X
authors.lastname: Zhang
authors.affiliation: The First Affiliated Hospital of Soochow University, Soochow, China.
authors.forename: De-Pei
authors.initials: DP
authors.lastname: Wu
authors.affiliation: Peking University Institute of Hematology, Peking University People's Hospital, No. 11 Xizhimen South Street, Xicheng District, Beijing, 100044, People's Republic of China. huangxiaojun@bjmu.edu.cn.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. huangxiaojun@bjmu.edu.cn.|Peking-Tsinghua Center for Life Sciences, Beijing, China. huangxiaojun@bjmu.edu.cn.
authors.forename: Xiao-Jun
authors.initials: XJ
authors.lastname: Huang
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000150:complications / Q000276:immunology / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival / Q000276:immunology; D006086:Graft vs Host Disease / Q000276:immunology; D006238:Haploidy; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006648:Histocompatibility / Q000276:immunology*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012042:Registries; D015996:Survival Rate; D013997:Time Factors; D055815:Young Adult
keywords: Acquired severe aplastic anemia; Haploidentical transplantation; Upfront