Bone marrow transplantation in 107 patients with severe aplastic anemia using cyclophosphamide and thoraco-abdominal irradiation for conditioning: long-term follow-up

Blood. 1991 Nov 1;78(9):2451-5.

Abstract

Since 1980, 107 consecutive patients (pts) underwent bone marrow transplantation (BMT) for nonconstitutional severe aplastic anemia (SAA) at our institution. All received conditioning with Cytoxan (150 mg/kg) and thoraco-abdominal irradiation (6 Gy) from an HLA-identical sibling donor. Mean age was 19 years (5 to 46 years). Forty-nine pts had less than 0.2 x 10(9)/L PMN and 53 failed to respond to previous immunosuppressive therapy before BMT. Graft-versus-host disease (GVHD) prophylaxis consisted of methotrexate (22 pts), cyclosporine (52 pts), or both (33 pts). With a median follow-up of 45 months (12 to 120 months), overall actuarial survival was 68% (confidence interval 95%:9.7). Of 16 factors tested, five were shown to adversely influence survival by multivariate analysis: grade greater than or equal to 2 acute GVHD (relative risk [RR]: 5.5), prior immunosuppressive therapy (RR: 3.5), female as donor (RR: 2.4), nonidiopathic SAA (RR: 2), and more than 0.2 x 10(9)/L PMN AA (RR: 2). Because acute GVHD was the most potent factor for survival, we analysed risk factors for acute GVHD. By multivariate analysis, 2 of 14 factors tested were independent: male as recipient (RR: 3) and previous alloimmunization of the donor (RR: 4.3). On long-term follow-up, chronic GVHD was observed in 49 pts of 89 surviving more than 100 days (55%). Multivariate analysis showed that infection before transplant (RR: 1.3) and previous history of acute GVHD (RR: 1.8) were associated with an increased risk of chronic GVHD.

MeSH terms

  • Abdomen
  • Adolescent
  • Adult
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / surgery*
  • Bone Marrow Transplantation* / adverse effects
  • Child
  • Cyclophosphamide / therapeutic use*
  • Cyclosporine / therapeutic use
  • Drug Therapy, Combination
  • Female
  • Graft Survival
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / prevention & control
  • Humans
  • Immunosuppression Therapy*
  • Male
  • Methotrexate / therapeutic use
  • Middle Aged
  • Multivariate Analysis
  • Thorax

Substances

  • Cyclosporine
  • Cyclophosphamide
  • Methotrexate