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HLA-matched sibling bone marrow transplantation for β-thalassemia major. Blood 2011 Feb 03;117(5):1745-50 PMID: 21119108 PMCID: PMC3056598

Pubmed ID

21119108

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.

Author List

Sabloff M, Chandy M, Wang Z, Logan BR, Ghavamzadeh A, Li CK, Irfan SM, Bredeson CN, Cowan MJ, Gale RP, Hale GA, Horan J, Hongeng S, Eapen M, Walters MC

Authors

Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Brent R. Logan PhD Director, Professor in the Institute for Health and Equity department at Medical College of Wisconsin




Scopus

2-s2.0-79551639277   70 Citations

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Bone Marrow Transplantation
Child
Child, Preschool
Female
Graft Rejection
Graft Survival
Graft vs Host Disease
HLA Antigens
Humans
International Agencies
Male
Siblings
Survival Rate
Tissue Donors
Transplantation, Homologous
Treatment Outcome
Young Adult
beta-Thalassemia
jenkins-FCD Prod-300 626508253d14e4184314fb9f66322a03a5906796