PMID- 26125718
OWN - NLM
STAT- MEDLINE
DCOM- 20160329
LR  - 20150701
IS  - 1676-5680 (Electronic)
IS  - 1676-5680 (Linking)
VI  - 14
IP  - 2
DP  - 2015 May 18
TI  - Allogeneic hematopoietic stem cell transplantation in children with aplastic 
      anemia.
PG  - 5234-45
LID - 10.4238/2015.May.18.15 [doi]
AB  - The aim of this study was to prospectively investigate the efficacy and safety of 
      fully matched allogeneic hematopoietic stem cell transplants in children with 
      severe aplastic anemia in China. A total of twenty patients with severe aplastic 
      anemia were enrolled in our study. Thirteen cases underwent transplantation with 
      fully human leukocyte antigen (HLA)-matched, granulocyte-colony stimulating 
      factor (G-CSF)-primed bone marrow and peripheral blood stem cells (PBSCs) from 
      matching sibling donors. One patient received fully HLA-matched bone marrow from 
      an unrelated donor. Six patients received fully HLA-matched G-CSF-primed PBSCs 
      from unrelated donors. The conditioning regimen included fludarabine, 
      cyclophosphamide, and rabbit anti-thymocyte globulin. Graft-versus-host disease 
      prophylaxis was conducted with cyclosporin A and short-course methotrexate. The 
      median follow-up duration was 3.08 years (range, 0.83-8.41years). The median time 
      of neutrophil recovery (>0.5 x 10(9)/L) was 14 days (range, 10-20 days), and the 
      median time of platelet recovery (>20 x 10(9)/L) was 19 days (range, 14-31 days). 
      The survival rate at the cutoff point of follow-up was 95.0% (19/20). Initial 
      engraftment rate was 95% (19/20). Late graft failure (graft failures occurring 1 
      year or longer after transplantation) was observed in one patient. Only one 
      patient developed Grade I acute graft-versus-host disease. Two cases suffered 
      from Epstein- Barr virus (EBV)-associated post-transplant lymphoproliferative 
      disorder and remitted after treatment with rituximab. One patient was diagnosed 
      with hyperthyroidism 2.5 years after transplantation. Our study indicated that 
      allogeneic hematopoietic stem cell transplantation is an effective and safe 
      treatment for children with severe aplastic anemia in China.
FAU - Xue, H-M
AU  - Xue HM
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Xu, H-G
AU  - Xu HG
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Huang, K
AU  - Huang K
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Guo, H-X
AU  - Guo HX
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Li, Y
AU  - Li Y
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Zhou, D-H
AU  - Zhou DH
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Huang, S-L
AU  - Huang SL
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Fang, J-P
AU  - Fang JP
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China.
FAU - Chen, C
AU  - Chen C
AD  - Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, 
      Guangzhou, China chunchendoc@126.com.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20150518
PL  - Brazil
TA  - Genet Mol Res
JT  - Genetics and molecular research : GMR
JID - 101169387
RN  - 0 (Antilymphocyte Serum)
RN  - 0 (HLA Antigens)
RN  - 143011-72-7 (Granulocyte Colony-Stimulating Factor)
RN  - 83HN0GTJ6D (Cyclosporine)
SB  - IM
MH  - Adolescent
MH  - Anemia, Aplastic/immunology/pathology/*therapy
MH  - Animals
MH  - Antilymphocyte Serum/administration & dosage/immunology
MH  - *Bone Marrow Transplantation
MH  - Child
MH  - Child, Preschool
MH  - China
MH  - Cyclosporine/administration & dosage
MH  - Female
MH  - Graft vs Host Disease/etiology/pathology
MH  - Granulocyte Colony-Stimulating Factor/administration & dosage
MH  - HLA Antigens/genetics/immunology
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Male
MH  - Rabbits
MH  - *Transplantation, Homologous
EDAT- 2015/07/01 06:00
MHDA- 2016/03/30 06:00
CRDT- 2015/07/01 06:00
PHST- 2015/07/01 06:00 [entrez]
PHST- 2015/07/01 06:00 [pubmed]
PHST- 2016/03/30 06:00 [medline]
AID - gmr5201 [pii]
AID - 10.4238/2015.May.18.15 [doi]
PST - epublish
SO  - Genet Mol Res. 2015 May 18;14(2):5234-45. doi: 10.4238/2015.May.18.15.