Cyclosporin A and short-term methotrexate versus cyclosporin A as graft versus host disease prophylaxis in patients with severe aplastic anemia given allogeneic bone marrow transplantation from an HLA-identical sibling: results of a GITMO/EBMT randomized trial

F Locatelli, B Bruno, M Zecca, M T Van-Lint, S McCann, W Arcese, S Dallorso, P Di Bartolomeo, F Fagioli, A Locasciulli, M Lawler, A Bacigalupo, Mark Lawler

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Abstract

A randomized trial was carried out comparing cyclosporin A (CsA) and short-term methotrexate (MTX) versus CsA alone for graft versus host disease (GVHD) prophylaxis in patients with severe aplastic anemia (SAA) undergoing allogeneic bone marrow transplantation (BMT) from a compatible sibling. Seventy-one patients (median age, 19 years; range, 4-46 years) were randomized to receive either CsA and MTX or CsA alone for the first 3 weeks after BMT. Subsequently, both groups received CsA orally, with gradual drug reduction until discontinuation 8 to 12 months after BMT. Patients randomized in both arms had comparable characteristics and received the same preparative regimen (ie, cyclophosphamide 200 mg/kg over 4 days). The median time for neutrophil engraftment was 17 days (range, 11-31 days) and 12 days (range, 4-45 days) for patients in the CsA/MTX group and the CsA alone group, respectively (P =.01). No significant difference was observed in the probability of either grade 2, grade 3, or grade 4 acute GVHD or chronic GVHD developing in the 2 groups. The Kaplan-Meier estimates of 1-year transplantation-related mortality rates for patients given either CsA/MTX or CsA alone were 3% and 15%, respectively (P =.07). With a median follow-up of 48 months from BMT, the 5-year survival probability is 94% for patients in the CsA/MTX group and 78% for those in the CsA alone group (P =. 05). These data indicate that the use of CsA with MTX is associated with improved survival in patients with SAA who receive transplants from compatible siblings. (Blood. 2000;96:1690-1697)

Original languageEnglish
Pages (from-to)1690-7
Number of pages8
JournalBlood
Volume96
Issue number5
Publication statusPublished - 01 Sep 2000

Keywords

  • Adolescent
  • Adult
  • Anemia, Aplastic
  • Bone Marrow Transplantation
  • Child
  • Child, Preschool
  • Cyclosporine
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Graft Rejection
  • Graft vs Host Disease
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents
  • Male
  • Methotrexate
  • Middle Aged
  • Nuclear Family
  • Survival Analysis
  • Time Factors
  • Transplantation, Homologous
  • Treatment Outcome

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