PMID- 30071526
OWN - NLM
STAT- MEDLINE
DCOM- 20190610
LR  - 20190613
IS  - 1421-9662 (Electronic)
IS  - 0001-5792 (Linking)
VI  - 140
IP  - 1
DP  - 2018
TI  - Transplant Outcomes in Beta-Thalassemia Major Patients Receiving Combined 
      Granulocyte Colony-Stimulating Factor-Primed Bone Marrow and Cord Blood Graft 
      Compared to Granulocyte Colony-Stimulating Factor-Primed Bone Marrow Alone.
PG  - 20-29
LID - 10.1159/000490407 [doi]
AB  - BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is currently the only 
      curative treatment for thalassemia majorTM. Graft rejection (GR) and 
      graft-versus-host disease (GVHD) are the primary obstacles to a successful 
      outcome. METHODS: We conducted a retrospective study of HSCT in 29 children 
      (median age at transplantation: 6 years) with Beta-thalassemia (beta-TM) after the 
      combined infusion of granulocyte colony-stimulating factor-primed bone marrow 
      (G-BM) and cord blood (CB) from the human leukocyte antigen (HLA)-identical 
      sibling donors. We also compared the outcomes of the co-transplanted children 
      with those of children with beta-TM who received G-BM alone from an HLA-identical 
      sibling donor (n = 26). RESULTS: Compared to the G-BM transplant (G-BMT) 
      recipients, those who received a co-transplant had a lower incidence of grade >/=II 
      acute (17.24 vs. 30.7%, p = 0.047) and limited chronic (0 vs.15.4%, p = 0.022) 
      GVHD as well as a lower incidence of GR (0 vs. 7.7%, p = 0.132). Neutrophil 
      recovery time was faster in the co-transplant group (18.5 vs. 21 days, p = 0.04). 
      All the patients were monitored until December 31, 2016; the median follow-up 
      time was 74 months<unterline>,</unterline> and the 5-year thalassemia-free 
      survival rate was 89.7% in the co-transplant group and 84.6% in the G-BMT-alone 
      group (p = 0.590). CONCLUSIONS: A combined CB and G-BM graft from an 
      HLA-identical sibling donor is an effective treatment option for TM in children, 
      with less acute and chronic GVHD.
CI  - (c) 2018 S. Karger AG, Basel.
FAU - Wen, Jianyun
AU  - Wen J
FAU - Haque, Qareen
AU  - Haque Q
FAU - Pei, Fuyu
AU  - Pei F
FAU - Chen, Libai
AU  - Chen L
FAU - Ruan, Yongsheng
AU  - Ruan Y
FAU - Liu, Xuan
AU  - Liu X
FAU - He, Yuelin
AU  - He Y
FAU - Feng, Xiaoqin
AU  - Feng X
FAU - Li, Chunfu
AU  - Li C
FAU - Wu, Xuedong
AU  - Wu X
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20180802
PL  - Switzerland
TA  - Acta Haematol
JT  - Acta haematologica
JID - 0141053
RN  - 0 (HLA Antigens)
RN  - 0 (Immunosuppressive Agents)
RN  - 143011-72-7 (Granulocyte Colony-Stimulating Factor)
SB  - IM
MH  - Adolescent
MH  - Bone Marrow Cells/cytology/drug effects
MH  - Cell Differentiation/*drug effects
MH  - Child
MH  - Child, Preschool
MH  - Cytomegalovirus/physiology
MH  - Disease-Free Survival
MH  - Fetal Blood/cytology/drug effects/metabolism
MH  - Graft vs Host Disease/etiology/pathology/prevention & control
MH  - Granulocyte Colony-Stimulating Factor/*pharmacology
MH  - HLA Antigens/immunology
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - Hematopoietic Stem Cells/cytology
MH  - Humans
MH  - Immunosuppressive Agents/therapeutic use
MH  - Infant
MH  - Retrospective Studies
MH  - Severity of Illness Index
MH  - Virus Activation
MH  - beta-Thalassemia/mortality/pathology/*therapy
OTO - NOTNLM
OT  - Beta-thalassemia major
OT  - Bone marrow
OT  - Cord blood
OT  - Hematopoietic stem cell transplant
EDAT- 2018/08/03 06:00
MHDA- 2019/06/14 06:00
CRDT- 2018/08/03 06:00
PHST- 2017/05/22 00:00 [received]
PHST- 2018/05/20 00:00 [accepted]
PHST- 2018/08/03 06:00 [pubmed]
PHST- 2019/06/14 06:00 [medline]
PHST- 2018/08/03 06:00 [entrez]
AID - 000490407 [pii]
AID - 10.1159/000490407 [doi]
PST - ppublish
SO  - Acta Haematol. 2018;140(1):20-29. doi: 10.1159/000490407. Epub 2018 Aug 2.