PMID- 2591400
OWN - NLM
STAT- MEDLINE
DCOM- 19900119
LR  - 20190813
IS  - 0340-6199 (Print)
IS  - 0340-6199 (Linking)
VI  - 149
IP  - 2
DP  - 1989 Nov
TI  - HLA-haploidentical bone marrow transplantation in three infants with adenosine 
      deaminase deficiency: stable immunological reconstitution and reversal of 
      skeletal abnormalities.
PG  - 104-9
AB  - Three infants with severe combined immunodeficiency and adenosine deaminase (ADA) 
      deficiency were treated by T-cell depleted bone marrow transplantation (BMT), 
      using human leukocyte antigen (HLA)-haploidentical parents as donors. In the 
      first patient, two initial transplants failed to engraft and no change of the 
      immunodeficiency was observed. In order to overcome this graft resistance, 
      cytoreductive conditioning was used prior to a third transplant. In the other two 
      patients, similar conditioning was used prior to initial transplants. In all 
      three patients, complete and permanent immunological reconstitution was observed 
      and they survive from 3.5 to 5 years after transplantation. In biopsies obtained 
      from iliac bones prior to BMT, osteochondral abnormalities characteristic of 
      ADA-deficiency were noted in all three patients. After successful 
      transplantation, these abnormalities had completely resolved. Our results 
      demonstrate that cytoreductive conditioning prior to HLA-haploidentical BMT is 
      useful in order to obtain stable engraftment and reversal of abnormalities 
      associated with ADA deficiency.
FAU - Bluetters-Sawatzki, R
AU  - Bluetters-Sawatzki R
AD  - Kinderklinik, Ulm, Federal Republic of Germany.
FAU - Friedrich, W
AU  - Friedrich W
FAU - Ebell, W
AU  - Ebell W
FAU - Vetter, U
AU  - Vetter U
FAU - Stoess, H
AU  - Stoess H
FAU - Goldmann, S F
AU  - Goldmann SF
FAU - Kleihauer, E
AU  - Kleihauer E
LA  - eng
PT  - Case Reports
PT  - Journal Article
PL  - Germany
TA  - Eur J Pediatr
JT  - European journal of pediatrics
JID - 7603873
RN  - 0 (HLA Antigens)
RN  - 0 (Immunosuppressive Agents)
RN  - EC 3.5.4.- (Nucleoside Deaminases)
RN  - EC 3.5.4.4 (Adenosine Deaminase)
SB  - IM
MH  - Adenosine Deaminase/*deficiency
MH  - *Bone Marrow Transplantation
MH  - Bone and Bones/drug effects/ultrastructure
MH  - Female
MH  - HLA Antigens
MH  - Humans
MH  - Immunologic Deficiency Syndromes/complications/*therapy
MH  - Immunosuppressive Agents/therapeutic use
MH  - Infant
MH  - Infant, Newborn
MH  - Lymphocyte Depletion
MH  - Male
MH  - Nucleoside Deaminases/*deficiency
EDAT- 1989/11/01 00:00
MHDA- 1989/11/01 00:01
CRDT- 1989/11/01 00:00
PHST- 1989/11/01 00:00 [pubmed]
PHST- 1989/11/01 00:01 [medline]
PHST- 1989/11/01 00:00 [entrez]
AID - 10.1007/BF01995857 [doi]
PST - ppublish
SO  - Eur J Pediatr. 1989 Nov;149(2):104-9. doi: 10.1007/BF01995857.