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Title: Outcomes of Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplantation by Using a Standard Myeloablative versus a Novel Reduced-Toxicity Conditioning Regimen According to a New Risk Stratification
Authors: Usanarat Anurathapan
Samart Pakakasama
Pimsiri Mekjaruskul
Nongnuch Sirachainan
Duantida Songdej
Ampaiwan Chuansumrit
Pimlak Charoenkwan
Arunee Jetsrisuparb
Kleebsabai Sanpakit
Bunchoo Pongtanakul
Piya Rujkijyanont
Arunotai Meekaewkunchorn
Rosarin Sruamsiri
Artit Ungkanont
Surapol Issaragrisil
Borje S. Andersson
Suradej Hongeng
Mahidol University
Maharaj Nakorn Chiang Mai Hospital
Khon Kaen University
Phramongkutklao College of Medicine
Queen Sirikit National Institute of Child Health
Naresuan University
University of Texas MD Anderson Cancer Center
Keywords: Medicine
Issue Date: 1-Jan-2014
Citation: Biology of Blood and Marrow Transplantation. Vol.20, No.12 (2014), 2066-2071
Abstract: © 2014 American Society for Blood and Marrow Transplantation. Improving outcomes among class 3 thalassemia patients receiving allogeneic hematopoietic stem cell transplantations (HSCT) remains a challenge. Before HSCT, patients who were ≥ 7 years old and had a liver size ≥ 5 cm constitute what the Center for International Blood and Marrow Transplant Research defined as a very high-risk subset of a conventional high-risk class 3 group (here referred to as class 3 HR). We performed HSCT in 98 patients with related and unrelated donor stem cells. Seventy-six of the patients with age < 10 years received the more conventional myeloablative conditioning (MAC) regimen (cyclophosphamide, busulfan, ± fludarabine); the remaining 22 patients with age ≥ 10 years and hepatomegaly (class 3 HR), and in several instances additional comorbidity problems, underwent HSCT with a novel reduced-toxicity conditioning (RTC) regimen (fludarabine and busulfan). We then compared the outcomes between these 2 groups (MAC versus RTC). Event-free survival (86% versus 90%) and overall survival (95% versus 90%) were not significantly different between the respective groups; however, there was a higher incidence of serious treatment-related complications in the MAC group, and although we experienced 6 graft failures in the MAC group (8%), there were none in the RTC group. Based on these results, we suggest that (1) class 3 HR thalassemia patients can safely receive HSCT with our novel RTC regimen and achieve the same excellent outcome as low/standard-risk thalassemia patients who received the standard MAC regimen, and further, (2) that this novel RTC approach should be tested in the low/standard-risk patient population.
ISSN: 15236536
Appears in Collections:Scopus 2011-2015

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