Haematologica (May 2007)

Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient

  • Sébastien Maury,
  • Marie-Lorraine Balère-Appert,
  • Zina Chir,
  • Jean-Michel Boiron,
  • Claire Galambrun,
  • Karima Yakouben,
  • Pierre Bordigoni,
  • Aude Marie-Cardine,
  • Noel Milpied,
  • Judith Kanold,
  • Natacha Maillard,
  • Gérard Socié,
  • Gérard Socié on behalf of the French Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)

DOI
https://doi.org/10.3324/haematol.10899
Journal volume & issue
Vol. 92, no. 5

Abstract

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Background and Objectives Severe acquired aplastic anemia (SAA) is a potentially fatal bone marrow failure syndrome occurring mainly in children and young adults. Immunosuppressive regimens and hematopoietic stem cell transplantation (HSCT) are the only two available curative treatments. Patients who lack an HLA-identical sibling donor may receive HSCT from an unrelated donor, a strategy historically associated with high mortality rates. Thus, for patients refractory to immunosuppressive regimens, the decision to transplant stem cells from unrelated donors is weighed against supportive care and often represents a dilemma for physicians. We aimed to determine whether outcome after unrelated HSCT has improved in recent years and, if so, to determine the factors responsible for the improvement.Design and Methods We analyzed the outcome of 89 patients (median age 17 years, range 0–52) with acquired SAA undergoing HSCT from an unrelated donor between 1989 and 2004. Cases were consecutively reported to the French Registry (SFGM-TC) by 25 centers.Results Patients transplanted during two successive time-periods (1989–1998 and 1999–2004) had different 5-year survival probabilities (±95% confidence interval): 29±7% and 50±7%, respectively (p