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Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA Working Party

Published inHaematologica, vol. 95, no. 6, p. 976-982
Publication date2010
Abstract

BACKGROUND: We analyzed the outcome of 100 patients with acquired severe aplastic anemia undergoing an alternative donor transplant, after immune suppressive therapy had failed. DESIGN AND METHODS: As a conditioning regimen, patients received either a combination of fludarabine, cyclophosphamide, and antithymocyte globulin (n=52, median age 13 years) or this combination with the addition of low dose (2 Gy) total body irradiation (n=48, median age 27 years). RESULTS: With a median follow-up of 1665 and 765 days, the actuarial 5-year survival was 73% for the group that received fludarabine, cyclophosphamide, and antithymocyte globulin and 79% for the group given the conditioning regimen including total body irradiation. Acute graft-versus-host disease grade III-IV was seen in 18% and 7% of the groups, respectively. Graft failure was seen in 17 patients with an overall cumulative incidence of 17% in patients receiving conditioning with or without total body irradiation: 9 of these 17 patients survive in the long-term. The most significant predictor of survival was the interval between diagnosis and transplantation, with 5-year survival rates of 87% and 55% for patients grafted within 2 years of diagnosis and more than 2 years after diagnosis, respectively (P=0.0004). Major causes of death were graft failure (n=7), post-transplant-lymphoproliferative-disease (n=4) and graft-versus-host disease (n=4). CONCLUSIONS: This study confirms positive results of alternative donor transplants in patients with severe aplastic anemia, the best outcomes being achieved in patients grafted within 2 years of diagnosis. Prevention of rejection and Epstein-Barr virus reactivation may further improve these results.

Keywords
  • Adolescent
  • Adult
  • Anemia, Aplastic/drug therapy/mortality/*surgery
  • Antilymphocyte Serum/*therapeutic use
  • Bone Marrow Transplantation/*methods
  • Child
  • Child, Preschool
  • Cyclophosphamide/*therapeutic use
  • Europe
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease/drug therapy/mortality/prevention & control
  • Hematopoietic Stem Cell Transplantation/*methods
  • Humans
  • Living Donors
  • Male
  • Middle Aged
  • Retrospective Studies
  • Survival Rate/trends
  • Transplantation Conditioning/methods
  • Vidarabine/*analogs & derivatives/therapeutic use
  • Whole-Body Irradiation/methods
  • Young Adult
Citation (ISO format)
BACIGALUPO, Andrea et al. Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA Working Party. In: Haematologica, 2010, vol. 95, n° 6, p. 976–982. doi: 10.3324/haematol.2009.018267
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ISSN of the journal0390-6078
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