Unmanipulated haploidentical haematopoietic stem cell transplantation for children with severe aplastic anaemia.
Haploidentical haematopoietic stem cell transplantation (haplo-HSCT) used to be a third-line treatment option for childhood severe aplastic anaemia (SAA). We conducted this retrospective study of 36 children (38 transplants) who received haplo-HSCT from human leucocyte antigen (HLA)-mismatched related donors between July 2002 and November 2013 at five HSCT centres in China, including 17 cases that were 5/6 HLA matched (Group 1) and 21 that were 4/6 or 3/6 HLA matched (Group 2). Although patients in Group 2 had a higher incidence of grade II-IV acute graft-versus-host disease (57·9% vs. 5·9%, P = 0·001), they had similar rates of graft failure (5·3% vs. 5·9%, P = 0·742) and overall survival (80·8% vs. 93·8%, P = 0·234) as Group 1. Unmanipulated haplo-HSCT is an effective treatment for SAA children with satisfactory outcome of this cohort, especially in the 5/6 HLA-matched group. For patients in critical situations, such as unresponsive to immunosuppressive therapy, refractory infection and failing first HSCT, to bring forward the timing of haplo-HSCT is a feasible salvage strategy with better and faster donor accessibility.
Key Laboratory of Paediatric Haematology&Oncology Ministry of Health, Department of Haematology/Oncology, Shanghai Children's Medical Centre, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Hua
H
Zhu
BaYi Children's Hospital Affiliated to Beijing Military General Hospital, Beijing, China.
Rong Mu
RM
Luo
Navy General Hospital, Beijing, China.
Zuo
Z
Luan
Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, China.
Vincent
V
Lee
West China Second University Hospital of Sichuan University, Sichuan, China.
Yi Ping
YP
Zhu
Key Laboratory of Paediatric Haematology&Oncology Ministry of Health, Department of Haematology/Oncology, Shanghai Children's Medical Centre, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Cheng Juan
CJ
Luo
Navy General Hospital, Beijing, China.
Xiang Feng
XF
Tang
BaYi Children's Hospital Affiliated to Beijing Military General Hospital, Beijing, China.
Ying Jian
YJ
Si
Key Laboratory of Paediatric Haematology&Oncology Ministry of Health, Department of Haematology/Oncology, Shanghai Children's Medical Centre, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Jing
J
Chen
D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D001782:Blood Donors; D002648:Child; D002675:Child, Preschool; D002681:China; D005260:Female; D006084:Graft Rejection / Q000276:immunology; D006086:Graft vs Host Disease / Q000276:immunology; D006680:HLA Antigens / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006648:Histocompatibility / Q000276:immunology*; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate
D006680:HLA Antigens
haematopoietic stem cell transplantation; haploidentical donor; paediatric; severe aplastic anaemia