PMID- 32798657
OWN - NLM
STAT- MEDLINE
DCOM- 20210623
LR  - 20210623
IS  - 1523-6536 (Electronic)
IS  - 1083-8791 (Linking)
VI  - 26
IP  - 11
DP  - 2020 Nov
TI  - Prospective Study of Allogeneic Hematopoietic Stem Cell Transplantation with 
      Post-Transplantation Cyclophosphamide and Antithymocyte Globulin from 
      HLA-Mismatched Related Donors for Nonmalignant Diseases.
PG  - e286-e291
LID - S1083-8791(20)30502-4 [pii]
LID - 10.1016/j.bbmt.2020.08.008 [doi]
AB  - Allogeneic hematopoietic stem cell transplantation (HSCT) is performed as a 
      curative treatment for children with nonmalignant diseases, such as bone marrow 
      failure syndromes and primary immunodeficiencies. Because 
      graft-versus-host-disease (GVHD) is a major factor affecting survival probability 
      and quality of life after HSCT, the availability of HLA-matched donors restricts 
      the application of HSCT. Recently, HSCT with post-transplantation 
      cyclophosphamide (PTCy) has emerged as a potent method to prevent GVHD after HSCT 
      from HLA-haploidentical donors, and some studies have suggested the safety of 
      PTCy-HSCT for nonmalignant diseases. We conducted a prospective clinical trial 
      aiming to help confirm the safety of HSCT and further reduction of GVHD using a 
      combination of PTCy and low-dose antithymocyte globulin (ATG) from HLA-mismatched 
      related donors for children with nonmalignant diseases. Six patients underwent 
      HSCT and achieved engraftment at a median of 14.5 days, and no patient developed 
      severe acute GVHD. All patients had sustained donor chimerism without developing 
      chronic GVHD at the last follow-up. In conclusion, HSCT with PTCy and low-dose 
      ATG from an HLA-mismatched related donor were feasible to control GVHD for 
      nonmalignant diseases in the children involved in our study. (c) 2020 American 
      Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.
CI  - Copyright (c) 2020 American Society for Transplantation and Cellular Therapy. 
      Published by Elsevier Inc. All rights reserved.
FAU - Osumi, Tomoo
AU  - Osumi T
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan; Department of Pediatric Hematology and Oncology Research, National 
      Research Institute for Child Health and Development, Tokyo, Japan.
FAU - Yoshimura, Satoshi
AU  - Yoshimura S
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Sako, Mayumi
AU  - Sako M
AD  - Department of Clinical Research Promotion, Clinical Research Center, National 
      Center for Child Health and Development, Tokyo, Japan.
FAU - Uchiyama, Toru
AU  - Uchiyama T
AD  - Department of Human Genetics, National Research Institute for Child Health and 
      Development, Tokyo, Japan.
FAU - Ishikawa, Takashi
AU  - Ishikawa T
AD  - Division of Immunology, National Center for Child Health and Development, Tokyo, 
      Japan.
FAU - Kawai, Toshinao
AU  - Kawai T
AD  - Division of Immunology, National Center for Child Health and Development, Tokyo, 
      Japan.
FAU - Inoue, Eisuke
AU  - Inoue E
AD  - Showa University Research Administration Center, Showa University, Tokyo, Japan.
FAU - Takimoto, Tetsuya
AU  - Takimoto T
AD  - Department of Childhood Cancer Data Management, National Center for Child Health 
      and Development, Tokyo, Japan.
FAU - Takeuchi, Ichiro
AU  - Takeuchi I
AD  - Center for Pediatric Inflammatory Bowel Disease, Division of Gastroenterology, 
      National Center for Child Health and Development, Tokyo, Japan.
FAU - Yamada, Masaki
AU  - Yamada M
AD  - Depertment of Advanced Medicine for Virus Infections, National Center for Child 
      Health and Development, Tokyo, Japan.
FAU - Sakamoto, Kenichi
AU  - Sakamoto K
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Yoshida, Kaoru
AU  - Yoshida K
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan; Department of Pediatric Hematology and Oncology Research, National 
      Research Institute for Child Health and Development, Tokyo, Japan.
FAU - Kimura, Yui
AU  - Kimura Y
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Matsukawa, Yukihiro
AU  - Matsukawa Y
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Matsumoto, Kana
AU  - Matsumoto K
AD  - Department of Clinical Pharmaceutics, Faculty of Pharmaceutical Sciences, 
      Doshisha Women's College of Liberal Arts, Kyoto, Japan.
FAU - Imadome, Ken-Ichi
AU  - Imadome KI
AD  - Depertment of Advanced Medicine for Virus Infections, National Center for Child 
      Health and Development, Tokyo, Japan.
FAU - Arai, Katsuhiro
AU  - Arai K
AD  - Center for Pediatric Inflammatory Bowel Disease, Division of Gastroenterology, 
      National Center for Child Health and Development, Tokyo, Japan.
FAU - Deguchi, Takao
AU  - Deguchi T
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Imai, Kohsuke
AU  - Imai K
AD  - Department of Community Pediatrics, Perinatal and Maternal Medicine, Graduate 
      School of Medical and Dental Sciences, Tokyo Medical and Dental University, 
      Tokyo, Japan.
FAU - Yuza, Yuki
AU  - Yuza Y
AD  - Department of Hematology and Oncology, Tokyo Metropolitan Children's Medical 
      Center, Tokyo, Japan.
FAU - Matsumoto, Kimikazu
AU  - Matsumoto K
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Onodera, Masafumi
AU  - Onodera M
AD  - Department of Human Genetics, National Research Institute for Child Health and 
      Development, Tokyo, Japan; Division of Immunology, National Center for Child 
      Health and Development, Tokyo, Japan.
FAU - Kanegane, Hirokazu
AU  - Kanegane H
AD  - Department of Child Health and Development, Graduate School of Medical and Dental 
      Sciences, Tokyo Medical and Dental University, Tokyo, Japan.
FAU - Tomizawa, Daisuke
AU  - Tomizawa D
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan.
FAU - Kato, Motohiro
AU  - Kato M
AD  - Children's Cancer Center, National Center for Child Health and Development, 
      Tokyo, Japan; Department of Pediatric Hematology and Oncology Research, National 
      Research Institute for Child Health and Development, Tokyo, Japan. Electronic 
      address: katom-tky@umin.ac.jp.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20200814
PL  - United States
TA  - Biol Blood Marrow Transplant
JT  - Biology of blood and marrow transplantation : journal of the American Society for 
      Blood and Marrow Transplantation
JID - 9600628
RN  - 0 (Antilymphocyte Serum)
RN  - 8N3DW7272P (Cyclophosphamide)
SB  - IM
MH  - Antilymphocyte Serum/therapeutic use
MH  - Child
MH  - Cyclophosphamide/therapeutic use
MH  - *Graft vs Host Disease/prevention & control
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Prospective Studies
MH  - Quality of Life
MH  - Transplantation Conditioning
OTO - NOTNLM
OT  - Graft-versus-host disease
OT  - Haploidentical donor
OT  - Nonmalignant disease
OT  - Post-transplantation cyclophosphamide
OT  - Prospective trial
EDAT- 2020/08/18 06:00
MHDA- 2021/06/24 06:00
CRDT- 2020/08/18 06:00
PHST- 2020/06/20 00:00 [received]
PHST- 2020/07/27 00:00 [revised]
PHST- 2020/08/05 00:00 [accepted]
PHST- 2020/08/18 06:00 [pubmed]
PHST- 2021/06/24 06:00 [medline]
PHST- 2020/08/18 06:00 [entrez]
AID - S1083-8791(20)30502-4 [pii]
AID - 10.1016/j.bbmt.2020.08.008 [doi]
PST - ppublish
SO  - Biol Blood Marrow Transplant. 2020 Nov;26(11):e286-e291. doi: 
      10.1016/j.bbmt.2020.08.008. Epub 2020 Aug 14.