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

Br J Haematol. 2001 Sep;114(3):706-11. doi: 10.1046/j.1365-2141.2001.02992.x.

Abstract

We report a favourable outcome in 15 patients with severe aplastic anaemia (SAA) who were < 20 years of age and who underwent bone marrow transplantation (BMT) from a human leucocyte antigen (HLA)-matched unrelated donor. All patients were non-responders to intensive immunosuppressive therapy (IST) and were multiply transfused. The conditioning regimen consisted of cyclophosphamide (60 mg/kg/d, on d -4 and -3), anti-thymocyte 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-versus-host disease (GVHD), 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 transplantation (median follow-up, 51 months). Moderate to severe acute GVHD occurred in 5 out of 15 patients (33%); only one patient developed extensive chronic GVHD. Considering our encouraging results, unrelated donor transplantation for SAA is recommended as a salvage therapy in non-responders to IST.

MeSH terms

  • Adolescent
  • Anemia, Aplastic / therapy*
  • Antilymphocyte Serum / administration & dosage
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Cyclophosphamide / administration & dosage
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease / therapy
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Male
  • Salvage Therapy*
  • T-Lymphocytes / immunology
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous
  • Whole-Body Irradiation

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclophosphamide