title: Cyclophosphamide/antithymocyte globulin conditioning of patients with severe aplastic anemia for marrow transplantation from HLA-matched siblings: preliminary results.
abstract: Many approaches have been taken to reducing the rate of graft failure and the incidence of graft-versus-host disease (GVHD) in bone marrow transplantation (BMT) of patients with severe aplastic anemia (SAA). The combination of cyclophosphamide with irradiation has had unequivocal success in reconstituting a sustained engraftment, but this procedure has severe associated risks such as second malignancies. Recently, cyclophosphamide (CYC) plus antithymocyte globulin (ATG) has been shown to be an effective alternative to irradiation-based programs in retransplants. Based on these experiences, the current clinical trial was started to prepare patients suffering from SAA for marrow transplantation from HLA-identical siblings with ATG plus CYC. Nine patients have been enrolled into the study so far. They received a total dose of 200 mg/kg CYC and concomitantly 120 mg/kg or 90 mg/kg ATG, followed by cyclosporine plus methotrexate as post-transplantation GVHD prophylaxis. Eight of nine patients survived without any transplant-associated complications; i.e., they had a documented stable engraftment without rejection and without acute or chronic GVHD. One patient died due to an Aspergillus sepsis prior to a definite engraftment. Although our data are preliminary because of the small number of patients enrolled and a follow-up of only 30 months, CYC plus ATG appears to be an effective preparative regimen for BMT in patients with SAA, resulting in a favorable outcome.
authors.affiliation: Center for Bone Marrow Transplantation, University Hospital Eppendorf, Hamburg, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Horstmann
authors.forename: M
authors.initials: M
authors.lastname: Stockschläder
authors.forename: W
authors.initials: W
authors.lastname: Krüger
authors.forename: M
authors.initials: M
authors.lastname: Hoffknecht
authors.forename: R
authors.initials: R
authors.lastname: Betker
authors.forename: H
authors.initials: H
authors.lastname: Kabisch
authors.forename: A
authors.initials: A
authors.lastname: Zander
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage / Q000627:therapeutic use*; D001228:Aspergillosis; D016026:Bone Marrow Transplantation; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use*; D016572:Cyclosporine / Q000008:administration & dosage / Q000627:therapeutic use; D005260:Female; D016469:Fungemia; D006086:Graft vs Host Disease / Q000517:prevention & control; D006680:HLA Antigens / Q000235:genetics*; D006648:Histocompatibility; D006801:Humans; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage / Q000627:therapeutic use; D012150:Polymorphism, Restriction Fragment Length
chemical_list: D000961:Antilymphocyte Serum; D006680:HLA Antigens; D016572:Cyclosporine; D003520:Cyclophosphamide; D008727:Methotrexate