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Thalassemia: Management after hematopoietic cell transplantation

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


Hematopoietic cell transplantation (HCT) is the only curative therapy for thalassemia. A number of potential post-transplant risks and complications must be addressed to ensure the best prognosis.

This topic discusses the care of patients with thalassemia who have undergone HCT, divided into two sections: completion of the transplant (eg, optimizing engraftment and immune status) and subsequent management, including assessment and therapy for excess iron stores; organ damage, which is often related to iron overload; and late complications of transplant such as chronic graft-versus-host disease (GVHD).

A general review of the treatment of thalassemia as well as indications for HCT, pretransplant evaluation, donor selection, stem cell source, conditioning regimen, and immediate post-transplant hematopoietic support are discussed in detail separately. (See "Management and prognosis of the thalassemias" and "Hematopoietic cell transplantation for transfusion-dependent thalassemia".)


Initial post-transplant management focuses on engraftment of the allogeneic hematopoietic cells and restoration of normal immune status. Important aspects of care include assessing and optimizing engraftment, treating infections, managing acute graft-versus-host disease (GVHD), and tapering immunosuppressive therapy.

Engraftment — For transplant to be effective, the donor hematopoietic cells need to engraft sufficiently to support hematopoiesis and non-thalassemic erythropoiesis. Engraftment is typically indicated by return of hematopoiesis in all three cell lines (white blood cells [WBCs], red blood cells [RBCs], and platelets) with decreasing need for transfusional support in the immediate post-transplant period.

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Literature review current through: Sep 2017. | This topic last updated: Sep 19, 2017.
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