Blood Journal
Leading the way in experimental and clinical research in hematology

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

  1. Mitchell Sabloff1,
  2. Mammen Chandy2,
  3. Zhiwei Wang3,
  4. Brent R. Logan3,
  5. Ardeshir Ghavamzadeh4,
  6. Chi-Kong Li5,
  7. Syed Mohammad Irfan6,
  8. Christopher N. Bredeson7,
  9. Morton J. Cowan8,
  10. Robert Peter Gale9,
  11. Gregory A. Hale10,
  12. John Horan11,
  13. Suradej Hongeng12,
  14. Mary Eapen3, and
  15. Mark C. Walters13
  1. 1Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, ON;
  2. 2Christian Medical College, Vellore, India;
  3. 3Medical College of Wisconsin, Center for International Blood and Marrow Transplant Research, Milwaukee, WI;
  4. 4Shariati Hospital, University of Tehran, Tehran, Iran;
  5. 5Chinese University of Hong Kong, Prince of Wales Hospital, Shatin, Hong Kong;
  6. 6National Institute of Blood Diseases and Bone Marrow Transplantation, Karachi, Pakistan;
  7. 7Medical College of Wisconsin, Milwaukee, WI;
  8. 8University of California San Francisco Medical Center, San Francisco, CA;
  9. 9Celgene Corporation, Summit, NJ;
  10. 10All Children's Hospital, St Petersburg, FL;
  11. 11Children's Healthcare of Atlanta at Egleston, Atlanta, GA;
  12. 12Ramathibodi Hospital Mahidol University, Bangkok, Thailand; and
  13. 13Children's Hospital & Research Center Oakland, Oakland, CA

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.

  • Submitted September 13, 2010.
  • Accepted November 16, 2010.
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