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HLA-matched sibling bone marrow transplantation for β-thalassemia major.
- Source :
-
Blood [Blood] 2011 Feb 03; Vol. 117 (5), pp. 1745-50. Date of Electronic Publication: 2010 Nov 30. - Publication Year :
- 2011
-
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.
- Subjects :
- Adolescent
Adult
Child
Child, Preschool
Female
Graft Rejection
Humans
International Agencies
Male
Siblings
Survival Rate
Tissue Donors
Transplantation, Homologous
Treatment Outcome
Young Adult
beta-Thalassemia immunology
Bone Marrow Transplantation
Graft Survival
Graft vs Host Disease
HLA Antigens metabolism
beta-Thalassemia therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1528-0020
- Volume :
- 117
- Issue :
- 5
- Database :
- MEDLINE
- Journal :
- Blood
- Publication Type :
- Academic Journal
- Accession number :
- 21119108
- Full Text :
- https://doi.org/10.1182/blood-2010-09-306829