title: Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study.
abstract: We conducted a prospective, multicentre study to confirm the feasibility of haplo-identical transplantation in treatment of severe aplastic anaemia (SAA) as salvage therapy, by analysing the outcomes of 101 patients who received haplo-identical transplantation between June 2012 and October 2015. All cases surviving for more than 28 d achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) days and 15 (range, 7-101) days for platelets, with a cumulative platelet engraftment incidence of 94·1 ± 0·1%. With a median follow-up of 18·3 (3·0-43·6) months, recipients from haplo-identical transplantation had more cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD, 33·7% vs. 4·2%, P < 0·001), more chronic GVHD (22·4% vs. 6·6%, P = 0·014) at 1 year, but similar grade III-IV aGVHD (7·9% vs. 2·1%, P = 0·157), 3-year estimated overall survival (OS, 89·0% vs. 91·0%, P = 0·555) and failure-free survival (FFS, 86·8% vs. 80·3%, P = 0·659) when compared with 48 patients who received contemporaneous transplantation from matched related donors. Multivariate analysis showed no significant difference in engraftment and survival between the two cohorts. Both OS and FFS for the entire population correlated significantly with grades III-IV aGVHD. In conclusion, haplo-identical transplantation is a feasible choice for SAA with favourable outcomes.
authors.affiliation: Peking University People's Hospital, Peking University Institute of Haematology, Beijing, China.
authors.forename: Lan-Ping
authors.initials: LP
authors.lastname: Xu
authors.affiliation: Guangzhou First People's Hospital, Guangzhou, China.
authors.forename: Shun-Qing
authors.initials: SQ
authors.lastname: Wang
authors.affiliation: The First affiliated Hospital of Soochow University, Soochow, China.
authors.forename: De-Pei
authors.initials: DP
authors.lastname: Wu
authors.affiliation: Changhai Hospital affiliated to Second Military Medical University, Shanghai, China.
authors.forename: Jian-Min
authors.initials: JM
authors.lastname: Wang
authors.affiliation: The First Hospital of Jilin University, Changchun, China.
authors.forename: Su-Jun
authors.initials: SJ
authors.lastname: Gao
authors.affiliation: The First affiliated Hospital of Xinjiang Medical University, Urumchi, China.
authors.forename: Ming
authors.initials: M
authors.lastname: Jiang
authors.affiliation: Lanzhou Military Area General Hospital, Lanzhou, China.
authors.forename: Cun-Bang
authors.initials: CB
authors.lastname: Wang
authors.affiliation: Xinqiao Hospital affiliated to Third Military Medical University, Chongqing, China.
authors.forename: Xi
authors.initials: X
authors.lastname: Zhang
authors.affiliation: Nanfang Hospital affiliated to Southern Medical University, Guangzhou, China.
authors.forename: Qi-Fa
authors.initials: QF
authors.lastname: Liu
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: Shandong Provincial Hospital, Jinan, China.
authors.forename: Xin
authors.initials: X
authors.lastname: Wang
authors.affiliation: Peking University People's Hospital, Peking University Institute of Haematology, Beijing, China. xjhrm@medmail.com.cn.|Beijing Key Laboratory of Haematopoietic Stem Cell Transplantation, Beijing, China. xjhrm@medmail.com.cn.|Peking-Tsinghua Centre for Life Sciences, Beijing, China. xjhrm@medmail.com.cn.
authors.forename: Xiao-Jun
authors.initials: XJ
authors.lastname: Huang
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000175:diagnosis* / Q000209:etiology / Q000401:mortality / Q000628:therapy*; D015415:Biomarkers; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000209:etiology; D006680:HLA Antigens / Q000235:genetics; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D019233:Retreatment; D012720:Severity of Illness Index; D016019:Survival Analysis; D014019:Tissue Donors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D015415:Biomarkers; D006680:HLA Antigens
keywords: haematopoietic stem cell transplantation; haploidentical; severe aplastic anaemia