Cyclophosphamide/antithymocyte globulin conditioning of patients with severe aplastic anemia for marrow transplantation from HLA-matched siblings : preliminary results

• Published in: Annals of hematology Vol. 71; no. 2; pp. 77 - 81
• Main Authors: HORSTMANN, M, STOCKSCHLÄDER, M, KRÜGER, W, HOFFKNECHT, M, BETKER, R, KABISCH, H, ZANDER, A
• Format: Journal Article
• Language: English
• Published: Berlin Springer 01-08-1995
• Subjects: Adolescent; Adult; Anemia, Aplastic - therapy; Antilymphocyte Serum - administration & dosage; Antilymphocyte Serum - therapeutic use; Aspergillosis; Biological and medical sciences; Bone Marrow Transplantation; Child; Cyclophosphamide - administration & dosage; Cyclophosphamide - therapeutic use; Cyclosporine - administration & dosage; Cyclosporine - therapeutic use; Female; Fungemia; Graft vs Host Disease - prevention & control; Histocompatibility; HLA Antigens - genetics; Humans; Immunomodulators; Male; Medical sciences; Methotrexate - administration & dosage; Methotrexate - therapeutic use; Pharmacology. Drug treatments; Polymorphism, Restriction Fragment Length; Human; Immunopathology; Chemoprophylaxis; Transfusion; Unrelated donor; Aplastic anemia; Homograft; Graft versus host reaction; Hemopathy; Prevention; Treatment; Bone marrow; Complication; Graft; Immunosuppressive agent; Histocompatibility
• ISSN: 0939-5555; 1432-0584
• DOI: 10.1007/BF01699250

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.