HLA-matched sibling bone marrow transplantation for β-thalassemia major

Blood. 2011 Feb 3;117(5):1745-50. doi: 10.1182/blood-2010-09-306829. Epub 2010 Nov 30.

Abstract

We describe outcomes after human leukocyte antigen-matched sibling bone marrow transplantation (BMT) for 179 patients with β-thalassemia major. The median age at transplantation was 7 years and the median follow-up was 6 years. The distribution of Pesaro risk class I, II, and III categories was 2%, 42%, and 36%, respectively. The day 30 cumulative incidence of neutrophil recovery and day 100 platelet recovery were 90% and 86%, respectively. Seventeen patients had graft failure, which was fatal in 11. Six of 9 patients with graft failure are alive after a second transplantation. The day 100 probability of acute graft-versus-host disease and 5-year probability of chronic graft-versus-host disease was 38% and 13%, respectively. The 5-year probabilities of overall- and disease-free survival were 91% and 88%, respectively, for patients with Pesaro risk class II, and 64% and 62%, respectively, for Pesaro risk class III. In multivariate analysis, mortality risks were higher in patients 7 years of age and older and those with hepatomegaly before BMT. The leading causes of death were interstitial pneumonitis (n = 7), hemorrhage (n = 8), and veno-occlusive disease (n = 6). Proceeding to BMT in children younger than 7 years before development of end-organ damage, particularly in the liver, should improve results after BMT for β-thalassemia major.

Publication types

  • Multicenter Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Female
  • Graft Rejection
  • Graft Survival*
  • Graft vs Host Disease*
  • HLA Antigens / metabolism*
  • Humans
  • International Agencies
  • Male
  • Siblings
  • Survival Rate
  • Tissue Donors
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult
  • beta-Thalassemia / immunology
  • beta-Thalassemia / therapy*

Substances

  • HLA Antigens