title: Unrelated donor BMT for Wiskott-Aldrich syndrome.
abstract: 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.
authors.affiliation: Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital, Los Angeles, CA 90027.
authors.forename: C
authors.initials: C
authors.lastname: Lenarsky
authors.forename: K
authors.initials: K
authors.lastname: Weinberg
authors.forename: D B
authors.initials: DB
authors.lastname: Kohn
authors.forename: R
authors.initials: R
authors.lastname: Parkman
mesh_terms: 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*
chemical_list: D006680:HLA Antigens