Abstract
Bone marrow transplantation is the only therapeutic option that can eliminate thalassemic disease. Early results indicated that children in class 3 Lucarelli had a much worse outcome because of high nonrejection mortality and high rejection rate. We therefore tried to investigate a nonmyeloablative stem cell transplantation (NST) approach for such a disease in order to reduce mortality and rejection. We report here the case of successful NST in a 10-year-old girl who had class 3 Lucarelli β-thalassemia major. The conditioning regimen consisted of busulfan, fludarabine, antilymphocyte globulin and total lymphoid irradiation. Her GVHD prophylaxis included mycophenolate mofetil and cyclosporin. The patient had full donor engraftment without acute and chronic GVHD. She is now alive and well and remains disease-free 1 year after transplant.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Lucarelli G, Galimberti M, Polchi P et al. Bone marrow transplantation in patients with thalassemia New Engl J Med 1990 322: 417 421
Andreani M, Manna M, Lucarelli G et al. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia Blood 1996 87: 3494 3499
Champlin R, Khouri I, Kornblau S et al. Reinventing bone marrow transplantation: reducing toxicity using nonmyeloablative, preparative regimens and induction of graft-versus-malignancy Curr Opin Oncol 1999 11: 87 95
Spitzer TR, McAfee S, Sackstein R et al. Intentional induction of mixed chimerism and achievement of antitumor responses after nonmyeloablative conditioning therapy and HLA-matched donor bone marrow transplantation for refractory hematologic malignancies Biol Blood Marrow Transplant 2000 6: 309 320
Krishnamurti L, Blazar BR, Wagner JE et al. Bone marrow transplantation without myeloablation for sickle cell disease New Engl J Med 2001 344: 68
Gaziev D, Polchi P, Lucarelli G et al. Second marrow transplant for graft failure in patients with thalassemia Bone Marrow Transplant 1999 24: 1299 1306
Xun CQ, Thompson JS, Jennings CD et al. Effect of total body irradiation, busulfan-cyclophosphamide, or cyclophosphamide conditioning on inflammatory cytokine release and development of acute and chronic graft-versus-host disease in H-2-incompatible transplanted SCID mice Blood 1994 83: 2360 2367
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Hongeng, S., Chuansumrit, A., Hathirat, P. et al. Full chimerism in nonmyeloablative stem cell transplantation in a β-thalassemia major patient (class 3 Lucarelli). Bone Marrow Transplant 30, 409–410 (2002). https://doi.org/10.1038/sj.bmt.1703655
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1703655
Keywords
This article is cited by
-
Cost utility analysis of reduced intensity hematopoietic stem cell transplantation in adolescence and young adult with severe thalassemia compared to hypertransfusion and iron chelation program
BMC Health Services Research (2013)
-
Allogeneic stem cell transplantation from unrelated donor for class 3 β-thalassemia major using reduced-intensity conditioning regimen
Bone Marrow Transplantation (2006)
-
Complete substitution of cyclophosphamide by fludarabine and ATG in a busulfan-based preparative regimen for children and adolescents with β-thalassemia
Bone Marrow Transplantation (2005)
-
Outcome of transplantation with unrelated donor bone marrow in children with severe thalassaemia
Bone Marrow Transplantation (2004)
-
Nonmyeloablative stem cell transplantation with a haploidentical donor in a class 3 lucarelli severe thalassemia patient
Bone Marrow Transplantation (2004)
