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

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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 anAsper-gillus 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.

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Authors and Affiliations

  1. Center for Bone Marrow Transplantation, Medical Clinic, University Hospital Eppendorf, Martinistrasse 52, D-20246, Hamburg, Germany

    M. Horstmann, M. Stockschläder, W. Krüger, M. Hoffknecht, R. Betker, H. Kabisch & A. Zander

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  1. M. Horstmann
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  2. M. Stockschläder
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Horstmann, M., Stockschläder, M., Krüger, W. et al. Cyclophosphamide/antithymocyte globulin conditioning of patients with severe aplastic anemia for marrow transplantation from HLA-matched siblings: Preliminary results. Ann Hematol 71, 77–81 (1995). https://doi.org/10.1007/BF01699250

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  • DOI: https://doi.org/10.1007/BF01699250

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