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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.

Abstract
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.
AuthorsUsanarat 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
JournalBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 20 Issue 12 Pg. 2066-71 (Dec 2014) ISSN: 1523-6536 [Electronic] United States
PMID25064743 (Publication Type: Comparative Study, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
CopyrightCopyright © 2014 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
Chemical References
  • Myeloablative Agonists
  • Cyclophosphamide
  • Vidarabine
  • Busulfan
  • fludarabine
Topics
  • Adolescent
  • Adult
  • Allografts
  • Busulfan (administration & dosage)
  • Child
  • Child, Preschool
  • Cyclophosphamide (administration & dosage)
  • Disease-Free Survival
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Male
  • Myeloablative Agonists (administration & dosage)
  • Risk Factors
  • Survival Rate
  • Thalassemia (mortality, therapy)
  • Transplantation Conditioning (methods)
  • Vidarabine (administration & dosage, analogs & derivatives)

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