PMID- 8765621
OWN - NLM
STAT- MEDLINE
DCOM- 19960925
LR  - 20190630
IS  - 0022-3476 (Print)
IS  - 0022-3476 (Linking)
VI  - 129
IP  - 2
DP  - 1996 Aug
TI  - Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a 
      single center.
PG  - 238-44
AB  - We retrospectively analyzed the outcome of bone marrow transplantation (BMT) 
      performed in 26 patients with Wiskott-Aldrich syndrome (WAS) in one center. 
      Twenty-eight transplantation procedures were performed. Ten unselected patients 
      received unmanipulated marrow from a donor with genetically identical human 
      leukocyte antigen (HLA). Eight patients were cured and survive 1.5 to 16.5 years 
      after BMT. One patient successfully received a T-cell-depleted marrow from a 
      matched unrelated donor. Sixteen patients were selected to receive a related HLA 
      partially incompatible BMT because of the occurrence of life-threatening 
      complications from the WAS (i.e., refractory thrombocytopenia, autoimmunity 
      including vasculitis and sepsis). All but one received T-cell-depleted marrow 
      after a conditioning regimen of busulfan and cyclophosphamide. One patient had 
      two BMTs. Engraftment occurred in 12 of 17 attempts. The addition of monoclonal 
      antibodies to lymphocyte function-associated antigen-1 and CD2 molecules appeared 
      to improve engraftment. Six patients were long-term survivors, whereas others 
      died of viral infections (n = 7), among which Epstein-Barr virus-induced 
      B-lymphocyte proliferative disorder was predominant. Delay in development of full 
      T- and B-cell functions accounted for severe infectious complications. These 
      results confirm the excellent outcome of HLA genetically identical BMT in WAS, 
      whereas BMT from HLA partially incompatible donors should be strictly restricted 
      to patients with severe complications of WAS.
FAU - Ozsahin, H
AU  - Ozsahin H
AD  - Department of Pediatrics, Universitats Kinderklinik, Zurich, Switzerland.
FAU - Le Deist, F
AU  - Le Deist F
FAU - Benkerrou, M
AU  - Benkerrou M
FAU - Cavazzana-Calvo, M
AU  - Cavazzana-Calvo M
FAU - Gomez, L
AU  - Gomez L
FAU - Griscelli, C
AU  - Griscelli C
FAU - Blanche, S
AU  - Blanche S
FAU - Fischer, A
AU  - Fischer A
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - J Pediatr
JT  - The Journal of pediatrics
JID - 0375410
RN  - 0 (Antibodies, Monoclonal)
RN  - 0 (CD2 Antigens)
RN  - 0 (HLA Antigens)
RN  - 0 (Immunosuppressive Agents)
RN  - 0 (Lymphocyte Function-Associated Antigen-1)
RN  - 8N3DW7272P (Cyclophosphamide)
RN  - G1LN9045DK (Busulfan)
SB  - IM
MH  - Adolescent
MH  - Antibodies, Monoclonal/therapeutic use
MH  - Autoimmune Diseases/complications
MH  - B-Lymphocytes/immunology
MH  - *Bone Marrow Transplantation
MH  - Busulfan/therapeutic use
MH  - CD2 Antigens/therapeutic use
MH  - Child
MH  - Child, Preschool
MH  - Cyclophosphamide/therapeutic use
MH  - Graft Survival
MH  - HLA Antigens/genetics
MH  - Herpesviridae Infections
MH  - Herpesvirus 4, Human
MH  - Humans
MH  - Immunosuppressive Agents/therapeutic use
MH  - Infant
MH  - Lymphocyte Depletion
MH  - Lymphocyte Function-Associated Antigen-1/therapeutic use
MH  - Retrospective Studies
MH  - Sepsis/complications
MH  - Survival Rate
MH  - T-Lymphocytes/immunology
MH  - Thrombocytopenia/complications
MH  - Treatment Outcome
MH  - Vasculitis, Leukocytoclastic, Cutaneous/complications
MH  - Wiskott-Aldrich Syndrome/*therapy
EDAT- 1996/08/01 00:00
MHDA- 1996/08/01 00:01
CRDT- 1996/08/01 00:00
PHST- 1996/08/01 00:00 [pubmed]
PHST- 1996/08/01 00:01 [medline]
PHST- 1996/08/01 00:00 [entrez]
AID - S0022-3476(96)70248-2 [pii]
AID - 10.1016/s0022-3476(96)70248-2 [doi]
PST - ppublish
SO  - J Pediatr. 1996 Aug;129(2):238-44. doi: 10.1016/s0022-3476(96)70248-2.