Bone marrow transplantation for patients with acquired severe aplastic anemia using cyclophosphamide and antithymocyte globulin: the experience from a single center

Hematol J. 2003;4(3):208-13. doi: 10.1038/sj.thj.6200246.

Abstract

Between 1998 and 2001, 31 (24 male, 7 female) patients with severe aplastic anemia (SAA) and a median age of 19 years (range, 4-39 years) received an allogeneic bone marrow transplantation. Marrow donors were genotypically HLA-identical siblings in 30 cases and a monozygous twin in one case. The median time from diagnosis to bone marrow transplantation was 1 month (range, 0.5-5 months). Conditioning regimen consisted of cyclophosphamide (CY) combined with antithymocyte globulin (ATG), in all patients. For graft-versus-host disease (GvHD) prophylaxis, all patients received methotrexate and cyclosporin. A total of 84% of patients had sustained grafts, whereas 16% rejected grafts between 3 and 20 months after transplantation. Of the five rejecting patients, three are alive with successful second engraftments and two died from infections. Acute grade II-IV GvHD was seen in only 11% of patients. A limited chronic GvHD was seen in one patient. With a median follow-up of 18 months (range, 5-42 months), survival rate was 86% and Karnofsky score was at least 90%. This study confirms the high success rate of the CY/ATG regimen in SAA allografted from an HLA-identical sibling. Early and late graft failure remains a problem and may require modification of this regimen.

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / complications
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Antilymphocyte Serum / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Bone Marrow Transplantation / adverse effects*
  • Bone Marrow Transplantation / immunology
  • Bone Marrow Transplantation / mortality
  • Cause of Death
  • Child
  • Child, Preschool
  • Cyclophosphamide / therapeutic use
  • Female
  • Graft Survival
  • Graft vs Host Disease / pathology
  • Graft vs Host Disease / prevention & control
  • Humans
  • Male
  • Retrospective Studies
  • Siblings
  • Transplantation Conditioning / methods
  • Transplantation, Isogeneic
  • Treatment Outcome

Substances

  • Antilymphocyte Serum
  • Cyclophosphamide