HLA-matched sibling bone marrow transplantation for beta-thalassemia major
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AbstractWe describe outcomes after human leukocyte antigen-matched sibling bone marrow transplantation (BMT) for 179 patients with beta-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 beta-thalassemia major. (Blood. 2011; 117(5): 1745-1750)
All Author(s) ListSabloff M, Chandy M, Wang ZW, Logan BR, Ghavamzadeh A, Li CK, Irfan SM, Bredeson CN, Cowan MJ, Gale RP, Hale GA, Horan J, Hongeng S, Eapen M, Walters MC
Journal nameBlood
Year2011
Month2
Day3
Volume Number117
Issue Number5
PublisherAmerican Society of Hematology
Pages1745 - 1750
ISSN0006-4971
eISSN1528-0020
LanguagesEnglish-United Kingdom
Web of Science Subject CategoriesHematology; HEMATOLOGY

Last updated on 2020-25-11 at 00:15