title: Bone marrow transplantation for patients with acquired severe aplastic anemia using cyclophosphamide and antithymocyte globulin: the experience from a single center.
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.
authors.affiliation: Centre National de Greffe de Moelle Osseuse (CNGMO), rue Jebel Lakhdar, 1006 Bab Saadoun, Tunis, Tunisia. aabdelkefi@yahoo.fr
authors.forename: Abderrahman
authors.initials: A
authors.lastname: Abdelkefi
authors.forename: Tarek
authors.initials: T
authors.lastname: Ben Othman
authors.forename: Saloua
authors.initials: S
authors.lastname: Ladeb
authors.forename: Lamia
authors.initials: L
authors.lastname: Torjman
authors.forename: Mohamed
authors.initials: M
authors.lastname: Hsaïri
authors.forename: Abdeladhim
authors.initials: A
authors.lastname: Ben Abdeladhim
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000150:complications / Q000401:mortality / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000009:adverse effects* / Q000276:immunology / Q000401:mortality; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000473:pathology / Q000517:prevention & control; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D035781:Siblings; D019172:Transplantation Conditioning / Q000379:methods; D014185:Transplantation, Isogeneic; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D003520:Cyclophosphamide