Publication: Pretransplant Immunosuppression followed by Reduced-Toxicity Conditioning and Stem Cell Transplantation in High-Risk Thalassemia: A Safe Approach to Disease Control
Issued Date
2013-08-01
Resource Type
ISSN
15236536
10838791
10838791
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2-s2.0-84880435286
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Mahidol University
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SCOPUS
Bibliographic Citation
Biology of Blood and Marrow Transplantation. Vol.19, No.8 (2013), 1259-1262
Suggested Citation
Usanarat Anurathapan, Samart Pakakasama, Piya Rujkijyanont, Nongnuch Sirachainan, Duantida Songdej, Ampaiwan Chuansumrit, Somtawin Sirireung, Pimlak Charoenkwan, Arunee Jetsrisuparb, Surapol Issaragrisil, Artit Ungkanont, Rosarin Sruamsiri, Supanart Srisala, Borje S. Andersson, Suradej Hongeng Pretransplant Immunosuppression followed by Reduced-Toxicity Conditioning and Stem Cell Transplantation in High-Risk Thalassemia: A Safe Approach to Disease Control. Biology of Blood and Marrow Transplantation. Vol.19, No.8 (2013), 1259-1262. doi:10.1016/j.bbmt.2013.04.023 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/32243
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Title
Pretransplant Immunosuppression followed by Reduced-Toxicity Conditioning and Stem Cell Transplantation in High-Risk Thalassemia: A Safe Approach to Disease Control
Abstract
Patients with class 3 thalassemia with high-risk features for adverse events after high-dose chemotherapy with hematopoietic stem cell transplantation (HSCT) are difficult to treat, tending to either suffer serious toxicity or fail to establish stable graft function. We performed HSCT in 18 such patients age ≥7 years and hepatomegaly using a novel approach with pretransplant immunosuppression followed by a myeloablative reduced-toxicity conditioning regimen (fludarabine and i.v. busulfan [Flu-IV Bu]) and then HSCT. The median patient age was 14 years (range, 10 to 18 years). Before the Flu-IV Bu+ antithymocyte globulin conditioning regimen, all patients received 1 to 2 cycles of pretransplant immunosuppression with fludarabine and dexamethasone. Thirteen patients received a related donor graft, and 5 received an unrelated donor graft. An initial prompt engraftment of donor cells with full donor chimerism was observed in all 18 patients, but 2 patients developed secondary mixed chimerism that necessitated withdrawal of immunosuppression to achieve full donor chimerism. Two patients (11%) had acute grade III-IV graft-versus-host disease, and 5 patients had limited chronic graft-versus-host disease. The only treatment-related mortality was from infection, and with a median follow-up of 42 months (range, 4 to 75), the 5-year overall survival and thalassemia-free survival were 89%. We conclude that this novel sequential immunoablative pretransplantation conditioning program is safe and effective for patients with high-risk class 3 thalassemia exhibiting additional comorbidities. © 2013 American Society for Blood and Marrow Transplantation.