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

• Published in: The hematology journal : the official journal of the European Haematology Association Vol. 4; no. 3; pp. 208 - 213
• Main Authors: Abdelkefi, Abderrahman, Ben Othman, Tarek, Ladeb, Saloua, Torjman, Lamia, Hsaïri, Mohamed, Ben Abdeladhim, Abdeladhim
• Format: Journal Article
• Language: English
• Published: England 2003
• Subjects: 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
• ISSN: 1466-4860; 1476-5632
• DOI: 10.1038/sj.thj.6200246

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.