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Int. J. Mol. Sci. 2017, 18(11), 2472; https://doi.org/10.3390/ijms18112472

Is the Benefit–Risk Ratio for Patients with Transfusion-Dependent Thalassemia Treated by Unrelated Cord Blood Transplantation Favorable?

Division of Hematology and Oncology, Department of Pediatrics, Chang Gung University and Children’s Hospital, 5 Fu-Shin Street, Kwei-Shan, Taoyuan 33302, Taiwan
Received: 12 September 2017 / Revised: 2 November 2017 / Accepted: 17 November 2017 / Published: 20 November 2017
(This article belongs to the Special Issue Thalassemia in 2017)
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Abstract

Transfusion-dependent thalassemia (TDT) is an inherited disorder characterized by absent or defective production of α- or β-hemoglobin chains. If untreated, the disease invariably culminates in death in early infancy due to cardiac failure or overwhelming infection. Although there is clear evidence of good health-related quality of life and return to normal life style, the choice to undergo hematopoietic stem cell transplantation (HSCT) remains a challenge because of the potential risk of transplant-related mortality (TRM) in TDT. Successful hematopoietic stem cell transplantation may cure the hematological manifestations of TDT, but introduces risks of TRM and morbidity. The low incidence of graft-versus-host disease (GVHD) provides the major rationale for pursuing unrelated cord blood transplantation (CBT). Considerable evidence suggests a lower rate of recurrence after CBT than after transplantation from adult donors. As the TRM, overall survival, and thalassemia-free survival for CBT improve, the utility of this stem cell source will expand to indications that have hitherto rarely used unrelated CBT. This paper summarizes the current progress in understanding the advances in unrelated CBT for thalassemia. Although as yet only in a limited number of patients, the results of unrelated CBT for thalassemia are encouraging. View Full-Text
Keywords: transfusion-dependent thalassemia; hematopoietic stem cell transplantation; cord blood transplantation transfusion-dependent thalassemia; hematopoietic stem cell transplantation; cord blood transplantation
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Jaing, T.-H. Is the Benefit–Risk Ratio for Patients with Transfusion-Dependent Thalassemia Treated by Unrelated Cord Blood Transplantation Favorable? Int. J. Mol. Sci. 2017, 18, 2472.

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