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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
Authors: 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
Mahidol University
Phramongkutklao College of Medicine
Maharaj Nakorn Chiang Mai Hospital
Khon Kaen University
Naresuan University
University of Texas MD Anderson Cancer Center
Keywords: Medicine
Issue Date: 1-Aug-2013
Citation: Biology of Blood and Marrow Transplantation. Vol.19, No.8 (2013), 1259-1262
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.
ISSN: 15236536
Appears in Collections:Scopus 2011-2015

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