PMID- 11157502
OWN - NLM
STAT- MEDLINE
DCOM- 20010315
LR  - 20210216
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 97
IP  - 3
DP  - 2001 Feb 1
TI  - Full hematopoietic engraftment after allogeneic bone marrow transplantation 
      without cytoreduction in a child with severe combined immunodeficiency.
PG  - 809-11
AB  - Bone marrow transplantation (BMT) for severe combined immunodeficiency (SCID) 
      with human leukocyte antigen (HLA)-identical sibling donors but no 
      pretransplantation cytoreduction results in T-lymphocyte engraftment and 
      correction of immune dysfunction but not in full hematopoietic engraftment. A 
      case of a 17-month-old girl with adenosine deaminase (ADA) deficiency SCID in 
      whom full hematopoietic engraftment developed after BMT from her HLA-identical 
      sister is reported. No myeloablative or immunosuppressive therapy or 
      graft-versus-host disease (GVHD) prophylaxis was given. Mild acute and chronic 
      GVHD developed, her B- and T-cell functions became reconstituted, and she is well 
      almost 11 years after BMT. After BMT, repeated studies demonstrated: (1) Loss of 
      a recipient-specific chromosomal marker in peripheral blood leukocytes (PBLs) and 
      bone marrow, (2) conversion of recipient red blood cell antigens to donor type, 
      (3) conversion of recipient T-cell, B-cell, and granulocyte lineages to donor 
      origin by DNA analysis, and (4) increased ADA activity and metabolic correction 
      in red blood cells and PBLs.
FAU - Rubocki, R J
AU  - Rubocki RJ
AD  - Departments of Pediatric-Hematology/Oncology and Pathology and Microbiology, 
      University of Nebraska Medical Center, Omaha, NE, USA.
FAU - Parsa, J R
AU  - Parsa JR
FAU - Hershfield, M S
AU  - Hershfield MS
FAU - Sanger, W G
AU  - Sanger WG
FAU - Pirruccello, S J
AU  - Pirruccello SJ
FAU - Santisteban, I
AU  - Santisteban I
FAU - Gordon, B G
AU  - Gordon BG
FAU - Strandjord, S E
AU  - Strandjord SE
FAU - Warkentin, P I
AU  - Warkentin PI
FAU - Coccia, P F
AU  - Coccia PF
LA  - eng
GR  - DK20902/DK/NIDDK NIH HHS/United States
PT  - Case Reports
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
RN  - EC 3.5.4.4 (Adenosine Deaminase)
SB  - IM
MH  - Adenosine Deaminase/*deficiency/metabolism
MH  - Blood Cell Count
MH  - *Bone Marrow Transplantation
MH  - Erythrocytes/enzymology
MH  - Female
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Infant
MH  - Leukocytes, Mononuclear/enzymology
MH  - Severe Combined Immunodeficiency/blood/enzymology/*surgery
MH  - Transplantation, Homologous
EDAT- 2001/02/07 11:00
MHDA- 2001/03/17 10:01
CRDT- 2001/02/07 11:00
PHST- 2001/02/07 11:00 [pubmed]
PHST- 2001/03/17 10:01 [medline]
PHST- 2001/02/07 11:00 [entrez]
AID - S0006-4971(20)56410-0 [pii]
AID - 10.1182/blood.v97.3.809 [doi]
PST - ppublish
SO  - Blood. 2001 Feb 1;97(3):809-11. doi: 10.1182/blood.v97.3.809.