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Published on: September 6, 2017
HLA-matched sibling bone marrow transplantation for β-thalassemia major
Mitchell Sabloff1, Mammen Chandy, Zhiwei Wang
1Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Ontario, Canada.
Insights
Bone marrow transplantation (BMT) offers high survival rates for β-thalassemia major, especially in younger patients. Early BMT before organ damage improves outcomes and reduces mortality risks.
Area of Science:
- Hematology
- Immunology
- Pediatric Medicine
Background:
- β-thalassemia major is a severe inherited blood disorder requiring lifelong transfusions.
- Bone marrow transplantation (BMT) is a potential cure but carries risks.
- Human leukocyte antigen (HLA)-matched sibling BMT is a primary treatment option.
Purpose of the Study:
- To evaluate outcomes of HLA-matched sibling BMT in patients with β-thalassemia major.
- To identify factors influencing survival and complications.
- To establish benchmarks for BMT success in this population.
Main Methods:
- Retrospective analysis of 179 patients undergoing HLA-matched sibling BMT.
- Assessment of neutrophil and platelet recovery, graft failure, and graft-versus-host disease (GVHD).
- Survival analysis using Kaplan-Meier curves and multivariate analysis for risk factors.
Main Results:
- High rates of neutrophil (90%) and platelet (86%) recovery were observed.
- Graft failure occurred in 17 patients, with 11 fatalities; 6 survived after a second transplant.
- Five-year overall survival was 91% for Pesaro risk class II and 64% for class III.
- Mortality risks increased with age (≥7 years) and hepatomegaly prior to BMT.
Conclusions:
- HLA-matched sibling BMT can achieve high survival rates for β-thalassemia major.
- Early BMT in children younger than 7 years, before end-organ damage, is crucial for optimal outcomes.
- Managing complications like graft failure and GVHD is essential for improving BMT success.
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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