PMID- 36757045
OWN - NLM
STAT- MEDLINE
DCOM- 20230228
LR  - 20230918
IS  - 1536-3678 (Electronic)
IS  - 1077-4114 (Linking)
VI  - 45
IP  - 2
DP  - 2023 Mar 1
TI  - Successful TCRalphabeta/CD19-Depleted Hematopoietic Cell Transplantation for a Patient 
      With Artemis Deficiency.
PG  - e285-e289
LID - 10.1097/MPH.0000000000002522 [doi]
AB  - Artemis deficiency is characterized by DNA double-strand breaks repairing 
      dysfunction and increased sensitivity to ionizing radiation and alkylating 
      reagents. We describe the first successful case of T-cell receptor 
      [TCR]alphabeta/CD19-depleted hematopoietic cell transplantation [HCT] for Artemis 
      deficiency in Japan. A 6-month-old Korean boy was diagnosed with 
      Artemis-deficient severe combined immunodeficiency. He had no human leukocyte 
      antigen (HLA)-matched sibling or unrelated donor. Therefore, TCRalphabeta/CD19-depleted 
      HCT from his haploidentical mother was performed. Despite mixed chimerism in 
      whole blood, T cells achieved complete donor chimerism 6 months after HCT. 
      TCRalphabeta/CD19-depleted HCT could be an effective treatment for patients with 
      radiation-sensitive severe combined immunodeficiency.
CI  - Copyright (c) 2023 Wolters Kluwer Health, Inc. All rights reserved.
FAU - Tomomasa, Dan
AU  - Tomomasa D
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Isoda, Takeshi
AU  - Isoda T
AUID- ORCID: 0000-0003-3349-3705
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Mitsuiki, Noriko
AU  - Mitsuiki N
AUID- ORCID: 0000-0002-0128-6907
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Inoue, Kento
AU  - Inoue K
AUID- ORCID: 0000-0002-6912-0436
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Nishimura, Akira
AU  - Nishimura A
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Uda, Kazuhiro
AU  - Uda K
AUID- ORCID: 0000-0002-2262-6130
AD  - Division of Infectious Diseases, Tokyo Metropolitan Children's Medical Center, 
      Tokyo, Japan.
FAU - Uchiyama, Toru
AU  - Uchiyama T
AUID- ORCID: 0000-0001-9610-0900
AD  - Department of Human Genetics, National Research Institute for Child Health and 
      Development, Tokyo, Japan.
FAU - Yamashita, Motoi
AU  - Yamashita M
AUID- ORCID: 0000-0002-9814-1033
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Kamiya, Takahiro
AU  - Kamiya T
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Endo, Akifumi
AU  - Endo A
AUID- ORCID: 0000-0002-8788-4042
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Takagi, Masatoshi
AU  - Takagi M
AUID- ORCID: 0000-0002-7580-9184
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Imai, Kohsuke
AU  - Imai K
AUID- ORCID: 0000-0003-2132-8403
AD  - Department of Community Pediatrics, Perinatal and Maternal Medicine, Graduate 
      School of Medical and Dental Sciences, Tokyo Medical and Dental University 
      (TMDU), Tokyo, Japan.
FAU - Kajiwara, Michiko
AU  - Kajiwara M
AD  - Center for Transfusion Medicine and Cell Therapy, Tokyo Medical and Dental 
      University Hospital, Tokyo, Japan.
FAU - Cowan, Morton J
AU  - Cowan MJ
AUID- ORCID: 0000-0002-1088-8045
AD  - Allergy Immunology and Blood and Marrow Transplant Division, Benioff Children's 
      Hospital, University of California San Francisco, San Francisco, California.
FAU - Morio, Tomohiro
AU  - Morio T
AUID- ORCID: 0000-0002-9259-1025
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
FAU - Kanegane, Hirokazu
AU  - Kanegane H
AUID- ORCID: 0000-0002-8696-9378
AD  - Deparment of Child Health and Development, Graduate School of Medical and Dental 
      Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
LA  - eng
PT  - Case Reports
PT  - Journal Article
DEP - 20230206
PL  - United States
TA  - J Pediatr Hematol Oncol
JT  - Journal of pediatric hematology/oncology
JID - 9505928
RN  - 0 (Antigens, CD19)
RN  - 0 (Receptors, Antigen, T-Cell, alpha-beta)
SB  - IM
MH  - Humans
MH  - Infant
MH  - Male
MH  - Antigens, CD19
MH  - *Graft vs Host Disease/etiology
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects
MH  - Receptors, Antigen, T-Cell, alpha-beta
MH  - *Severe Combined Immunodeficiency/genetics
MH  - T-Lymphocytes
MH  - Transplantation Conditioning
MH  - Unrelated Donors
COIS- The authors declare no conflict of interest.
EDAT- 2023/02/10 06:00
MHDA- 2023/03/03 06:00
CRDT- 2023/02/09 07:03
PHST- 2022/03/10 00:00 [received]
PHST- 2022/06/12 00:00 [accepted]
PHST- 2023/02/10 06:00 [pubmed]
PHST- 2023/03/03 06:00 [medline]
PHST- 2023/02/09 07:03 [entrez]
AID - 00043426-202303000-00032 [pii]
AID - 10.1097/MPH.0000000000002522 [doi]
PST - ppublish
SO  - J Pediatr Hematol Oncol. 2023 Mar 1;45(2):e285-e289. doi: 
      10.1097/MPH.0000000000002522. Epub 2023 Feb 6.