Excellent long-term survival after allogeneic marrow transplantation in patients with severe aplastic anemia

Bone Marrow Transplant. 1997 Jun;19(12):1191-6. doi: 10.1038/sj.bmt.1700819.

Abstract

Between 1982 and 1996, 20 patients (10 male, 10 female) with severe aplastic anemia (SAA) with a median age of 25 years (17-37 years), received grafts from an HLA-identical sibling (n = 17), HLA-identical unrelated donor (n = 2) or identical twin (n = 1). The median time from diagnosis to marrow transplantation (BMT) was 15 months (range 1-96 months). More than half of the patients had received more than 10 units of red blood cells or platelet transfusions prior to BMT. Pretransplant immunosuppression consisted of cyclophosphamide (CY) alone (n = 10), CY in combination with total body irradiation (n = 8), and CY and antithymocyte globulin (n = 2). For graft-versus-host disease (GVHD) prophylaxis methotrexate (MTX) alone (n = 9) or MTX with cyclosporin A (n = 10) were given. One patient died on day 18 after marrow grafting due to infection; all other patients had complete and sustained engraftment (95%). Eight patients developed acute GVHD (42%), nine patients chronic GVHD (53%) including four with extensive disease manifestation. One patient experienced a secondary malignancy 11 years after BMT. Eighteen patients followed for a median of 9.45 years (0.42-14.7 years) have sustained hematological reconstitution and are alive and well with a Karnofsky performance score of at least 90%. Thus, excellent long-term survival and low morbidity make allogeneic or syngeneic BMT the treatment of choice for younger patients with severe aplastic anemia.

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Bone Marrow Transplantation* / adverse effects
  • Bone Marrow Transplantation* / immunology
  • Diseases in Twins
  • Family
  • Female
  • Follow-Up Studies
  • Graft Survival
  • Graft vs Host Disease / etiology
  • HLA Antigens
  • Humans
  • Living Donors
  • Male
  • Survival Rate
  • Transplantation, Homologous
  • Transplantation, Isogeneic
  • Twins, Monozygotic

Substances

  • HLA Antigens