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Specific issues related to hematopoietic cell transplantation in beta thalassemia

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


Patients with homozygous beta thalassemia require chronic blood transfusions in order to survive; however, these transfusions cause iron overload that is ultimately fatal if not continuously treated. Despite the first attempts at using gene therapy in humans, hematopoietic cell transplantation (HCT) is the only widely available curative therapy for this disorder.

With regard to HCT, patients with beta thalassemia have features that make clinical management different from that of patients with leukemia. These include:

Tissue damage, especially hepatic, related to iron overload

Absence of previous chemotherapy and immunosuppression

Absence of a malignant clone


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Literature review current through: Jul 2017. | This topic last updated: Jun 23, 2015.
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