Unrelated donor bone marrow transplantation to treat severe aplastic anaemia in children and young adults

Br J Haematol. 1996 Jul;94(1):65-72. doi: 10.1046/j.1365-2141.1996.d01-1772.x.

Abstract

Alternative donor bone marrow transplantation (BMT) to treat severe aplastic anaemia (SAA) in children and young adults has been complicated by high rates of graft rejection and severe graft-versus-host disease (GVHD). We hypothesized that increased immunosuppression combined with T-cell depletion of the marrow graft would enable successful use of unrelated donor BMT in this disease. Preconditioning consisted of cytosine arabinoside, cyclophosphamide, and total body irradiation (TBI). T-cell depletion was with the anti-CD3 antibody T10B9. GVHD prophylaxis consisted of cyclosporine A. 28 previously transfused patients were transplanted. Nine donor/recipient pairs were HLA matched. As of 1 January 1996, 15/28 (54%) patients are alive, transfusion independent and well with a range of follow-up of 13 months to 8 years (median 2.75 years). Fatalities include all three patients with nonengraftment and all three patients with grade III/IV GVHD. Other fatalities were due to infections or therapy-related toxicity. The incidence >or= grade II acute GVHD was 28%. These data show that in children with SAA who have failed immunosuppression, unrelated donor BMT offers a reasonable hope of long-term survival.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / therapy*
  • Bone Marrow Transplantation / adverse effects
  • Bone Marrow Transplantation / methods*
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Graft Survival
  • Graft vs Host Disease / etiology
  • Humans
  • Infant
  • Male
  • Survival Analysis
  • Treatment Outcome