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

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

Mitchell Sabloff  1 , Mammen Chandy, Zhiwei Wang, Brent R Logan, Ardeshir Ghavamzadeh, Chi-Kong Li, Syed Mohammad Irfan, Christopher N Bredeson, Morton J Cowan, Robert Peter Gale, Gregory A Hale, John Horan, Suradej Hongeng, Mary Eapen, Mark C Walters
Affiliations
Multicenter Study

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

Mitchell Sabloff et al. Blood. .

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.

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Figures

Figure 1
Figure 1
Probability of OS by age and hepatomegaly.
Figure 2
Figure 2
Probability of DFS by age and hepatomegaly.
Figure 3
Figure 3
Probability of OS by Pesaro risk class.
See this image and copyright information in PMC

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