Unrelated donor BMT for Wiskott-Aldrich syndrome.
The role of allogeneic sibling BMT for children with Wiskott-Aldrich syndrome is established. Mismatched T cell-depleted BMT has been successful, although significant problems with graft rejection, GVHD, and post-transplant lymphoproliferative disorders have been reported. We have performed four BMTs for children with Wiskott-Aldrich syndrome utilizing phenotypically HLA-identical unrelated donors. A non-TBI (total body irradiation) conditioning regimen was utilized, and BM was not T cell-depleted. All patients engrafted and developed significant, although manageable, GVHD. All patients are alive 3+ to 17+ months post-transplant. These results suggest that matched unrelated donor BMT has a definite role in the treatment of Wiskott-Aldrich syndrome.
Lenarsky
C
C
Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital, Los Angeles, CA 90027.
Weinberg
K
K
Kohn
D B
DB
Parkman
R
R
D000208:Acute Disease; D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D003131:Combined Modality Therapy; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000209:etiology; D006680:HLA Antigens; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D016896:Treatment Outcome; D014923:Wiskott-Aldrich Syndrome / Q000188:drug therapy / Q000628:therapy*
D006680:HLA Antigens