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Hematopoietic cell transplantation for transfusion-dependent thalassemia

Author
Emanuele Angelucci, MD
Section Editor
Robert S Negrin, MD
Deputy Editor
Jennifer S Tirnauer, MD

INTRODUCTION

Hematopoietic cell transplantation (HCT) is the only available curative therapy for thalassemia, but HCT carries many risks and costs, and many individuals will not have access to HCT due to lack of a suitable donor and/or financial constraints.

This topic discusses the use of HCT in patients with transfusion-dependent thalassemia (eg, beta thalassemia major), including predictors of a successful outcome, pretransplant evaluation, donor selection, and source of stem cells, as well as management issues in the immediate post-transplant period.

Care following transplant, including donor chimerism (ie, engraftment), graft failure, retransplantation, chronic graft-versus-host disease, and reduction of excess iron stores, is discussed in detail separately. (See "Thalassemia: Management after hematopoietic cell transplantation" and "Iron chelators: Choice of agent, dosing, and adverse effects".)

The general management of thalassemia, including the decision to pursue medical therapy or transplantation, is also presented in detail separately. (See "Management and prognosis of the thalassemias".)

INDICATIONS AND PREDICTORS OF A GOOD OUTCOME

Allogeneic HCT is the only available curative therapy for thalassemia. However, HCT carries many risks and costs, and for many individuals a human leukocyte antigen (HLA)-identical sibling donor is not available. Thus, the decision to pursue HCT is challenging and must incorporate information about the patient's age, health, and resources as well as the values and preferences of the patient and family.

                             
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Literature review current through: Nov 2017. | This topic last updated: Sep 07, 2017.
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