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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.
AuthorsM Horstmann, M Stockschläder, W Krüger, M Hoffknecht, R Betker, H Kabisch, A Zander
JournalAnnals of hematology (Ann Hematol) Vol. 71 Issue 2 Pg. 77-81 (Aug 1995) ISSN: 0939-5555 [Print] Germany
PMID7654856 (Publication Type: Clinical Trial, Journal Article)
Chemical References
  • Antilymphocyte Serum
  • HLA Antigens
  • Cyclosporine
  • Cyclophosphamide
  • Methotrexate
Topics
  • Adolescent
  • Adult
  • Anemia, Aplastic (therapy)
  • Antilymphocyte Serum (administration & dosage, therapeutic use)
  • Aspergillosis
  • Bone Marrow Transplantation
  • Child
  • Cyclophosphamide (administration & dosage, therapeutic use)
  • Cyclosporine (administration & dosage, therapeutic use)
  • Female
  • Fungemia
  • Graft vs Host Disease (prevention & control)
  • HLA Antigens (genetics)
  • Histocompatibility
  • Humans
  • Male
  • Methotrexate (administration & dosage, therapeutic use)
  • Polymorphism, Restriction Fragment Length

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