Summary:
We conducted a study of unrelated donor bone marrow transplantation (BMT) in 11 children with severe thalassaemia. The conditioning regimen consisted of busulphan, cyclophosphamide and antilymphocyte globulin. All received T-cell nondepleted bone marrow. The median marrow-nucleated cell dose was 4.9 × 108 /kg (range; 3.5−8.0 × 108 /kg). Median time of granulocyte recovery was 16 days (range; 13–21 days), and of platelet recovery was 39 days (range; 14–196). Grade 2–4 acute graft-versus-host disease (GVHD) developed in six patients (54%), and grade 3–4 in one patient (9%). Three (27%) of 11 evaluable patients had chronic GVHD (limited stage). All 11 patients are alive without thalassaemia after a median follow-up time of 397 days (range; 171–814 days). This study lends support to consideration of unrelated donor BMT as an acceptable therapy to cure severe thalassaemia especially in patients who are young and do not yet show irreversible severe complications of iron overload.
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
Hongeng S, Krance RA, Bowman LC et al. Outcomes of transplantation with matched-sibling and unrelated-donor bone marrow in children with leukaemia. Lancet 1997; 350: 767–771.
Davies SM, Kollman C, Anasetti C et al. Engraftment and survival after unrelated-donor bone marrow transplantation: a report from National Marrow Donor Program. Blood 2000; 96: 4096–4121.
Contu L, La Nassa G, Arras M et al. Successful unrelated bone marrow transplantation in beta-thalassaemia. Bone Marrow Transplant 1994; 13: 329–331.
Lucarelli G, Giardini C, Angelucci E . Bone marrow transplantation in thalassemia. In: Winter JN, (ed). Blood Stem Cell Transplantation. Kluwer Academic: Boston, MA, 1997, pp 305–315.
Muretto P, Angelucci E, Del Fiasco S, Lucarelli G . Reversal features of hepatic haemosiderosis and haemochromatosis in thalassemia after bone marrow transplantation. Prog Clin Biol Res 1989; 309: 299–314.
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 2002; 30: 409–410.
Champlin RE, Schmitz N, Horowitz MM et al. Blood stem cells compared with bone marrow as a source of hematopoietic cells for allogeneic transplantation. Blood 2000; 95: 3702–3709.
Gaziev D, Galimberti M, Lucarelli G et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants. Bone Marrow Transplant 2000; 25: 815–821.
Sullivan KM, Anasetti C, Horowitz M et al. Unrelated and HLA-nonidentical related donor marrow transplantation for thalassemia and leukemia. Ann NY Acad Sci 1998; 850: 312–324.
La Nassa G, Giardini C, Argiolu F et al. Unrelated donor bone marrow transplantation for thalassemia: the effect of extended haplotypes. Blood 2002; 99: 4350–4356.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Hongeng, S., Pakakasama, S., Chaisiripoomkere, W. et al. Outcome of transplantation with unrelated donor bone marrow in children with severe thalassaemia. Bone Marrow Transplant 33, 377–379 (2004). https://doi.org/10.1038/sj.bmt.1704361
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1704361
Keywords
This article is cited by
-
Normalized levels of red blood cells expressing phosphatidylserine, their microparticles, and activated platelets in young patients with β-thalassemia following bone marrow transplantation
Annals of Hematology (2017)
-
Unrelated Umbilical Cord Blood Transplant for Children with β-Thalassemia Major
Indian Journal of Hematology and Blood Transfusion (2015)
-
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)
-
A cost-utility and budget impact analysis of allogeneic hematopoietic stem cell transplantation for severe thalassemic patients in Thailand
BMC Health Services Research (2010)
-
Quality of life in patients with transfusion-dependent thalassemia after hematopoietic SCT
Bone Marrow Transplantation (2008)