Thalassemia: Management after hematopoietic cell transplantation
- Emanuele Angelucci, MD
Emanuele Angelucci, MD
- Head, Hematology Department
- Cagliari Hospital, Italy
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".)
COMPLETION OF THE TRANSPLANT
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.To continue reading this article, you must log in with your personal, hospital, or group practice subscription. For more information on subscription options, click below on the option that best describes you:
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- COMPLETION OF THE TRANSPLANT
- - Full donor engraftment
- - Mixed chimerism
- - Graft failure
- - Overview of graft failure
- Thalassemia recurrence
- Marrow aplasia
- Graft-versus-host disease
- - Weaning immunosuppressive therapy
- - Acute GVHD
- - Chronic GVHD
- Other transplant toxicities
- LONG-TERM MANAGEMENT
- Iron stores
- - Estimation of iron stores
- - Targets for iron removal
- - Comparison of iron removal methods
- - Patients who can tolerate phlebotomy
- - Patients who require chelation
- Assessing and treating organ dysfunction
- - Liver disease
- - Heart disease
- - Endocrine dysfunction
- Quality of life
- SOCIETY GUIDELINE LINKS
- SUMMARY AND RECOMMENDATIONS