Allogenic stem cell transplantation for nonmalignant disorders using matched unrelated donors

We here report 25 patients with nonmalignant disorders, ie, severe aplastic anemia (SAA, n = 12) or inborn errors of metabolism (IEM, n = 13), who underwent allogeneic hematopoietic stem cell transplantation (HSCT) from unrelated high-resolution typed HLA-A, -B, and -DRbeta1 identical donors. One patient had an HLA-B subtype-mismatched donor. Conditioning for SAA mainly consisted of cyclophosphamide and total body irradiation, and that for IEM consisted of busulfan and cyclophosphamide. All patients received antithymocyte globulin during conditioning. After HSCT, they were given cyclosporine combined with methotrexate for immunosuppression. Two patients rejected their grafts: 1 died of pneumonia, and the other was successfully regrafted. The cumulative incidence of acute graft-versus-host disease grades II to IV was 24%, whereas chronic graft-versus-host disease occurred in 21%. The 5-year survival rates were 83% in the SAA group and 85% in those with IEM. We conclude that HSCT with HLA-A, -B, and -DRbeta1 genomically matched unrelated donors in combination with antithymocyte globulin in the conditioning regimen gives encouraging results in patients with SAA or IEM.