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.