BONE-MARROW TRANSPLANTATION IN 26 PATIENTS WITH WISKOTT-ALDRICH SYNDROME FROM A SINGLE-CENTER

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. T en unselected patients received unmanipulated marrow from a donor with genetically identical human leukocyte antigen (HLA). Eight patients w ere cured and survive 1.5 to 16.5 years after BMT, One patient success fully received a T-cell-depleted marrow from a matched unrelated donor , Sixteen patients were selected to receive a related HLA partially in compatible BMT because of the occurrence of life-threatening complicat ions from the WAS (i.e., refractory thrombocytopenia, autoimmunity inc luding vasculitis and sepsis), All but one received T-cell-depleted ma rrow after a conditioning regimen of busulfan and cyclophosphamide, On e patient had two BMTs, Engraftment occurred in 12 of 17 attempts. The addition of monoclonal antibodies to lymphocyte function-associated a ntigen-1 and CD2 molecules appeared to improve engraftment. Six patien ts were long-term survivors, whereas others died of viral infections ( n = 7), among which Epstein-Barr virus-induced B-lymphocyte proliferat ive disorder was predominant. Delay in development of full T- and B-ce ll functions accounted for severe infectious complications, These resu lts confirm the excellent outcome of HLA genetically identical BMT in WAS, whereas BMT from HLA partially incompatible donors should be stri ctly restricted to patients with severe complications of WAS.