HLA-matched sibling bone marrow transplantation for β-thalassemia major
- 3 February 2011
- journal article
- research article
- Published by American Society of Hematology in Blood
- Vol. 117 (5), 1745-1750
- https://doi.org/10.1182/blood-2010-09-306829
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.Keywords
This publication has 42 references indexed in Scilit:
- Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoringBlood, 2010
- Second bone marrow transplantation for patients with thalassemia: risks and benefitsHaematologica, 2009
- Clinical hemoglobinopathies: iron, lungs and new bloodCurrent Opinion in HIV and AIDS, 2007
- A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemiaBlood, 2006
- Randomized trial of two different conditioning regimens for bone marrow transplantation in thalassemia – the role of busulfan pharmacokinetics in determining outcomeBone Marrow Transplantation, 2005
- Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDSBlood, 2004
- Relationship of plasma pharmacokinetics of high-dose oral busulfan to the outcome of allogeneic bone marrow transplantation in children with thalassemiaBone Marrow Transplantation, 1997
- IBMTR Severity INDEX FOR GRADING ACUTE GRAFT‐VERSUS‐HOST DISEASE: RETROSPECTIVE COMPARISON WITH GLUCKSBERG GRADEBritish Journal of Haematology, 1997
- Marrow Transplantation in Patients with Thalassemia Responsive to Iron Chelation TherapyNew England Journal of Medicine, 1993
- Bone Marrow Transplantation in Patients with ThalassemiaNew England Journal of Medicine, 1990