Publication: Matched sibling donor hematopoietic stem cell transplantation for thalassemia
2
Issued Date
2016-11-01
Resource Type
ISSN
15317048
10656251
10656251
Other identifier(s)
2-s2.0-84981714779
Rights
Mahidol University
Rights Holder(s)
SCOPUS
Bibliographic Citation
Current Opinion in Hematology. Vol.23, No.6 (2016), 508-514
Suggested Citation
Surapol Issaragrisil, Chutima Kunacheewa Matched sibling donor hematopoietic stem cell transplantation for thalassemia. Current Opinion in Hematology. Vol.23, No.6 (2016), 508-514. doi:10.1097/MOH.0000000000000286 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/41077
Research Projects
Organizational Units
Authors
Journal Issue
Thesis
Title
Matched sibling donor hematopoietic stem cell transplantation for thalassemia
Author(s)
Other Contributor(s)
Abstract
© 2016 Wolters Kluwer Health, Inc. Purpose of review Bone marrow transplantation is the only curative treatment for severe thalassemia. Since its successful first report in 1981, more than 4000 patients with this disease worldwide underwent bone marrow transplantation. The purpose of this review is to update the most recent reports of matched sibling donor hematopoietic stem cell transplantation in thalassemia. Recent findings Advanced and improved transplant techniques result in the improved outcomes in those transplants from a matched sibling donor with transplant-related mortality less than 5%. Class 3 patients aged at least 7 years and liver enlargement at least 5 cm have a very high risk of graft rejection and regimen-related toxicity. This subset of patients require innovative approaches to overcome the morbidity and mortality. Those include the addition of hydroxyurea, azathioprine, and fludarabine as preconditioning to busulfan, thiotepa, and cyclophosphamide. Novel conditioning consisting of pretransplant immunosuppression with two cycles of fludarabine and dexamethasone followed by reduced intensity conditioning with fludarabine, busulfan, and thymoglobulin has been developed. Summary Bone marrow transplantation in young low-risk (class 1 and 2) patients should be performed as soon as possible. For class 3 severe thalassemia, novel conditioning regimens have been developed to overcome graft rejection and regimen-related toxicity. Hematopoietic stem cell transplantation in adults who have been well chelated should be offered with clinical trials.