PMID- 32800027
OWN - NLM
STAT- MEDLINE
DCOM- 20200925
LR  - 20200925
IS  - 1008-8830 (Print)
IS  - 1008-8830 (Linking)
VI  - 22
IP  - 8
DP  - 2020 Aug
TI  - [Peripheral blood stem cell transplantation from HLA-mismatched unrelated donor 
      or haploidentical donor for the treatment of X-linked agammaglobulinemia].
PG  - 821-827
AB  - Allogeneic stem cell transplantation (allo-SCT) is currently the only curative 
      option for patients with X-linked agammaglobulinemia (XLA). In this study, 
      patient 1 aged 4 years who underwent allogeneic peripheral blood stem cell 
      transplantation (allo-PBSCT) from HLA-mismatched unrelated donor; patient 2 aged 
      24 years (childhood onset) with primary cutaneous acral CD8(+) T cell lymphoma 
      who underwent allo-PBSCT from haploidentical relative donor. Both were treated by 
      reduced toxicity myeloablative conditioning with post-transplantation 
      cyclophosphamide (PTCy), anti-thymocyte globulin (ATG), methotrexate (MTX) and 
      cyclosporine (CsA) for graft-versus-host-disease (GVHD) prophylaxis. In patient 
      1, neutrophil and platelet engraftment were observed on day 11 
      post-transplantation; the donor chimerism dropped on day 90 post-transplantation, 
      and recovered on day 150 with donor lymphocyte infusion (DLI). In patient 2, 
      neutrophil and platelet engraftment were observed on days 20 and 87 
      post-transplantation respectively, with complete donor chimerism on day 30 
      post-transplantation. The serum levels of IgG, IgM and IgA and the percentage of 
      CD19(+) B cells in peripheral blood of patients 1 and 2 returned to normal within 
      2 months and more than 1 year after transplantation respectively. There was no 
      evidence of acute GVHD for the two patients. Patient 1 developed a limited type 
      of skin chronic GVHD after DLI, which disappeared after anti-GVHD treatment. This 
      is the first report of successful treatment for two XLA patients using PTCy with 
      allo-PBSCT from HLA-mismatched unrelated donor or haploidentical donor, combining 
      with improved conditioning, which expands the pool of eligible donors for 
      patients with XLA.
FAU - Nie, Ling
AU  - Nie L
AD  - Department of Hematology, Xiangya Hospital, Central South University, Changsha 
      410008, China. fu.bin@csu.edu.cn.
FAU - Su, Tao
AU  - Su T
FAU - Yang, Kai-Tai
AU  - Yang KT
FAU - Zhao, Liang
AU  - Zhao L
FAU - Hu, Jian
AU  - Hu J
FAU - Yang, Shuang-Hui
AU  - Yang SH
FAU - Xu, Ya-Jing
AU  - Xu YJ
FAU - Fu, Bin
AU  - Fu B
LA  - chi
PT  - Case Reports
PT  - Journal Article
PL  - China
TA  - Zhongguo Dang Dai Er Ke Za Zhi
JT  - Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
JID - 100909956
RN  - 0 (HLA Antigens)
RN  - Bruton type agammaglobulinemia
SB  - IM
MH  - Agammaglobulinemia/*therapy
MH  - Genetic Diseases, X-Linked/*therapy
MH  - *Graft vs Host Disease
MH  - HLA Antigens
MH  - Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - *Peripheral Blood Stem Cell Transplantation
MH  - Treatment Outcome
MH  - Unrelated Donors
MH  - Young Adult
PMC - PMC7441510
EDAT- 2020/08/18 06:00
MHDA- 2020/09/26 06:00
PMCR- 2020/08/15
CRDT- 2020/08/18 06:00
PHST- 2020/08/18 06:00 [entrez]
PHST- 2020/08/18 06:00 [pubmed]
PHST- 2020/09/26 06:00 [medline]
PHST- 2020/08/15 00:00 [pmc-release]
AID - 10.7499/j.issn.1008-8830.2006150 [pii]
AID - zgddekzz-22-8-821 [pii]
AID - 10.7499/j.issn.1008-8830.2006150 [doi]
PST - ppublish
SO  - Zhongguo Dang Dai Er Ke Za Zhi. 2020 Aug;22(8):821-827. doi: 
      10.7499/j.issn.1008-8830.2006150.