PMID- 36546031
OWN - NLM
STAT- MEDLINE
DCOM- 20221223
LR  - 20230105
IS  - 2095-9281 (Electronic)
IS  - 2095-9273 (Linking)
VI  - 67
IP  - 9
DP  - 2022 May 15
TI  - Long-term follow-up of haploidentical transplantation in relapsed/refractory 
      severe aplastic anemia: a multicenter prospective study.
PG  - 963-970
LID - S2095-9273(22)00039-1 [pii]
LID - 10.1016/j.scib.2022.01.024 [doi]
AB  - In recent decades, haploidentical stem cell transplantation (haplo-SCT) to treat 
      severe aplastic anemia (SAA) has achieved remarkable progress. However, long-term 
      results are still lacking. We conducted a multicenter prospective study involving 
      SAA patients who underwent haplo-SCT as salvage therapy. Long-term outcomes were 
      assessed, mainly focusing on survival and quality of life (QoL). Longitudinal QoL 
      was prospectively evaluated during pretransplantation and at 3 and 5 years 
      posttransplantation using the SF-36 scale in adults and the PedsQL 4.0 scale in 
      children. A total of 287 SAA patients were enrolled, and the median follow-up was 
      4.56 years (range, 3.01-9.05 years) among surviving patients. During the 
      long-term follow-up, 268 of 275 evaluable patients (97.5%) obtained sustained 
      full donor chimerism, and 93.4% had complete hematopoietic recovery. The 
      estimated overall survival and failure-free survival for the whole cohort at 
      9 years were 85.4% +/- 2.1% and 84.0% +/- 2.2%, respectively. Age (>/=18 years) and a 
      poorer performance status (ECOG >1) were identified as risk factors for survival 
      outcomes. For QoL recovery after haplo-SCT, we found that QoL progressively 
      improved from pretransplantation to the 3-year and 5-year time points with 
      statistical significance. The occurrence of chronic graft versus host disease was 
      a risk factor predicting poorer QoL scores in both the child and adult cohorts. 
      At the last follow-up, 74.0% of children and 72.9% of adults returned to normal 
      school or work. These inspiring long-term outcomes suggest that salvage 
      transplantation with haploidentical donors can be routine practice for SAA 
      patients without human leukocyte antigen (HLA)-matched donors.
CI  - Copyright (c) 2022 Science China Press. Published by Elsevier B.V. All rights 
      reserved.
FAU - Xu, Lan-Ping
AU  - Xu LP
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China; National Clinical Research Center for Hematologic Disease, 
      Beijing 100044, China.
FAU - Xu, Zheng-Li
AU  - Xu ZL
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China; National Clinical Research Center for Hematologic Disease, 
      Beijing 100044, China.
FAU - Wang, Shun-Qing
AU  - Wang SQ
AD  - Department of Hematology, Guangzhou First People's Hospital, Guangzhou 510180, 
      China.
FAU - Wu, De-Pei
AU  - Wu DP
AD  - Department of Hematology, The First Affiliated Hospital of Soochow University, 
      Soochow 215004, China.
FAU - Gao, Su-Jun
AU  - Gao SJ
AD  - Department of Hematology, The First Hospital of Jilin University, Changchun 
      130061, China.
FAU - Yang, Jian-Min
AU  - Yang JM
AD  - Department of Hematology, Changhai Hospital Affiliated to Second Military Medical 
      University, Shanghai 200433, China.
FAU - Xia, Ling-Hui
AU  - Xia LH
AD  - Department of Hematology, Xiehe Hospital Affiliated to Huazhong University of 
      Science and Technology, Wuhan 430022, China.
FAU - Liu, Qi-Fa
AU  - Liu QF
AD  - Department of Hematology, Nanfang Hospital Affiliated to Southern Medical 
      University, Guangzhou 510515, China.
FAU - Jiang, Ming
AU  - Jiang M
AD  - Department of Hematology, The First Affiliated Hospital of Xinjiang Medical 
      University, Urumchi 830054, China.
FAU - Bai, Hai
AU  - Bai H
AD  - Department of Hematology, General Hospital of Lanzhou Military Region of PLA, 
      Lanzhou 730050, China.
FAU - Zhang, Xi
AU  - Zhang X
AD  - Medical Center of Hematology, The Second Affiliated Hospital of Army Medical 
      University, Chongqing 400037, China.
FAU - Wang, Xin
AU  - Wang X
AD  - Department of Hematology, Shandong Provincial Hospital, Jinan 250021, China.
FAU - Huang, Xiao-Jun
AU  - Huang XJ
AD  - Peking University People's Hospital, Peking University Institute of Hematology, 
      Beijing 100044, China; National Clinical Research Center for Hematologic Disease, 
      Beijing 100044, China; Beijing Key Laboratory of Hematopoietic Stem Cell 
      Transplantation, Beijing 100044, China; Peking-Tsinghua Center for Life Sciences, 
      Beijing 100044, China. Electronic address: xjhrm@medmail.com.cn.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
DEP - 20220131
PL  - Netherlands
TA  - Sci Bull (Beijing)
JT  - Science bulletin
JID - 101655530
SB  - IM
MH  - Child
MH  - Adult
MH  - Humans
MH  - Adolescent
MH  - Transplantation, Haploidentical/methods
MH  - Follow-Up Studies
MH  - Prospective Studies
MH  - Quality of Life
MH  - *Anemia, Aplastic/therapy
MH  - *Graft vs Host Disease/etiology
OTO - NOTNLM
OT  - Aplastic anemia
OT  - Haploidentical
OT  - Long-term follow-up
OT  - Quality of life
EDAT- 2022/12/23 06:00
MHDA- 2022/12/24 06:00
CRDT- 2022/12/22 04:44
PHST- 2021/09/16 00:00 [received]
PHST- 2021/10/23 00:00 [revised]
PHST- 2022/01/18 00:00 [accepted]
PHST- 2022/12/22 04:44 [entrez]
PHST- 2022/12/23 06:00 [pubmed]
PHST- 2022/12/24 06:00 [medline]
AID - S2095-9273(22)00039-1 [pii]
AID - 10.1016/j.scib.2022.01.024 [doi]
PST - ppublish
SO  - Sci Bull (Beijing). 2022 May 15;67(9):963-970. doi: 10.1016/j.scib.2022.01.024. 
      Epub 2022 Jan 31.