Unrelated Bone Marrow Transplantation for a 19-Year-Old Boy with Wiskott-Aldrich Syndrome

Abstract

Wiskott-Aldrich syndrome (WAS) is an X-linked recessive immune deficiencysyndrome, and is characterized by a triad of thrombocytopenia with small platelet size, recurrent infection and eczema. Its novel gene was identified in 1994. At present, stem cell transplantation (SCT) is the only established therapy, and this is performed in the early stage if a HLA-matched donor is available. We report on a 19-year-old WAS patient who received a transplantation from an unrelated donor BMT (UD-BMT). He was the oldest WAS patient to undergo SCT in Japan. Inspite of severe complications, his SCT was successfully conducted. The usefulness of UD-BMT for WAS patients is discussed.