Improved survival following bone marrow transplantation for aplastic anaemia

Abstract

Patients (46) were severe aplastic anemia were transplanted with a new pretransplant immunosuppressive regimen consisting of cyclophosphamide (200 mg/kg) and low-dose total body irradiation (3 Gy). This regimen (Cy-TBI-2) was designed to decrease the high risk of graft rejection associated with the use of cyclophosphamide alone, without increasing the incidence of graft-vs.-host disease (GHVD) or interstitial pneumonia (IPn). Two-year actuarial disease-free survival of patients conditioned with CY-TBI-2 was 62% (95% CI: 47-77%). Only 1 patient rejected her graft and the incidence and severity of GVHD and IPn were not increased compared to previous studies. Patients < 25 yr of age had excellent 2-yr survival of 82% (95% CI: 69-95%). CY-TBI-2 apparently is an effective means of preventing graft-rejection and achieving long-term disease-free survival in multiply transfused patients with severe aplastic anemia.