Unrelated donor marrow transplantation in children with severe aplastic anaemia using cyclophosphamide, anti-thymocyte globulin and total body irradiation

We report a favourable outcome in 15 patients with severe aplastic anaemia (SAA) who were <20 years of age and who underwent bone marrow transplantati on (BMT) from a human leucocyte antigen (BLA)-matched unrelated donor. All patients were non-responders to intensive immunosuppressive therapy (IST) a nd were multiply transfused. The conditioning regimen consisted of cyclopho sphamide (60 mg/kg/d, on d -4 and -3), antithymocyte globulin (2.5 mg/kg/d, on d -5 to -2) and total body irradiation (2.5 Gy x 2/d, on d -2 and -1). Patients received cyclosporine and methotrexate for prophylaxis of graft-ve rsus-host disease (GVED), except for the last four who received tacrolimus instead of cyclosporine. Donor/recipient pairs were identical for HLA class I and II antigens by serological typing, but four pairs were found to have a mismatch at the HLA-A, -B or -DRB1 locus by high-resolution typing. All patients achieved rapid engraftment and are alive at 2-86 months after tran splantation (median follow-up, 51 months). Moderate to severe acute GVHD oc curred in 5 out of 15 patients (33%); only one patient developed extensive chronic GVHD. Considering our encouraging results, unrelated donor transpla ntation for SAA is recommended as a salvage therapy in nonresponders to IST .