PMID- 33000368
OWN - NLM
STAT- MEDLINE
DCOM- 20210618
LR  - 20210618
IS  - 1865-3774 (Electronic)
IS  - 0925-5710 (Linking)
VI  - 113
IP  - 2
DP  - 2021 Feb
TI  - Bone marrow transplantation from a human leukocyte antigen-mismatched unrelated 
      donor in a case with C1q deficiency associated with refractory systemic lupus 
      erythematosus.
PG  - 302-307
LID - 10.1007/s12185-020-03004-7 [doi]
AB  - Human C1q deficiency is frequently associated with systemic lupus erythematosus 
      (SLE), which requires long-term systemic corticosteroid administration. We report 
      the case of a 12-year-old female patient with C1q deficiency presenting with 
      intractable SLE who successfully underwent bone marrow transplantation from a 
      human leukocyte antigen (HLA)-mismatched unrelated donor with an 
      immunosuppressive conditioning regimen based on fludarabine, melphalan, and 
      anti-thymocyte globulin. She developed Grade I graft-versus-host disease, but did 
      not have any transplantation-related morbidity. Complete donor chimerism has been 
      maintained for 2 years after transplantation, leading to the restoration of C1q 
      levels and the resolution of SLE symptoms. Normal C1q mRNA expression was 
      observed in CD14 + cells. Hematopoietic stem cell transplantation from an 
      HLA-mismatched donor is a feasible treatment for patients with C1q deficiency 
      with refractory SLE that is dependent on systemic corticosteroid treatment who do 
      not have an HLA-matched donor.
FAU - Matsumura, Risa
AU  - Matsumura R
AUID- ORCID: 0000-0002-4871-928X
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan. rmats@hiroshima-u.ac.jp.
FAU - Mochizuki, Shinji
AU  - Mochizuki S
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
FAU - Maruyama, Natsuki
AU  - Maruyama N
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
FAU - Morishita, Yusuke
AU  - Morishita Y
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
FAU - Kawaguchi, Hiroshi
AU  - Kawaguchi H
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
FAU - Okada, Satoshi
AU  - Okada S
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
FAU - Tsumura, Miyuki
AU  - Tsumura M
AD  - Department of Pediatrics, Graduate School of Biomedical and Health Sciences, 
      Hiroshima University, Hiroshima, Japan.
FAU - Kaji, Shunsaku
AU  - Kaji S
AD  - Department of Pediatrics, Tsuyama Central Hospital, Tsuyama, Okayama, Japan.
FAU - Shimizu, Junya
AU  - Shimizu J
AD  - Department of Pediatrics, National Hospital Organization Okayama Medical Center, 
      Okayama, Japan.
FAU - Shimada, Akira
AU  - Shimada A
AD  - Department of Pediatric Hematology and Oncology, Okayama University Hospital, 
      Okayama, Japan.
FAU - Kobayashi, Masao
AU  - Kobayashi M
AD  - Department of Pediatrics, Hiroshima University Hospital, 1-2-3 Kasumi, Minami-ku, 
      Hiroshima, 734-8551, Japan.
LA  - eng
PT  - Case Reports
PT  - Journal Article
DEP - 20200930
PL  - Japan
TA  - Int J Hematol
JT  - International journal of hematology
JID - 9111627
RN  - 0 (HLA Antigens)
RN  - 80295-33-6 (Complement C1q)
SB  - IM
MH  - *Bone Marrow Transplantation/adverse effects/methods
MH  - Child
MH  - Complement C1q/*deficiency
MH  - Disease Management
MH  - Disease Susceptibility
MH  - Female
MH  - Graft vs Host Disease/drug therapy/etiology/prevention & control
MH  - HLA Antigens/genetics/immunology
MH  - Histocompatibility Testing
MH  - Humans
MH  - Lupus Erythematosus, Systemic/diagnosis/*etiology/*therapy
MH  - Transplantation Conditioning/methods
MH  - Transplantation, Homologous
MH  - Treatment Outcome
MH  - *Unrelated Donors
OTO - NOTNLM
OT  - C1q deficiency
OT  - Hematopoietic stem cell transplantation
OT  - Systemic lupus erythematosus
EDAT- 2020/10/02 06:00
MHDA- 2021/06/22 06:00
CRDT- 2020/10/01 05:40
PHST- 2020/05/29 00:00 [received]
PHST- 2020/09/10 00:00 [accepted]
PHST- 2020/08/10 00:00 [revised]
PHST- 2020/10/02 06:00 [pubmed]
PHST- 2021/06/22 06:00 [medline]
PHST- 2020/10/01 05:40 [entrez]
AID - 10.1007/s12185-020-03004-7 [pii]
AID - 10.1007/s12185-020-03004-7 [doi]
PST - ppublish
SO  - Int J Hematol. 2021 Feb;113(2):302-307. doi: 10.1007/s12185-020-03004-7. Epub 
      2020 Sep 30.