Specific issues related to hematopoietic cell transplantation in beta thalassemia
- Emanuele Angelucci, MD
Emanuele Angelucci, MD
- Head, Hematology Department
- Cagliari Hospital, Italy
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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- EVALUATION BEFORE TRANSPLANTATION
- ISSUES RELATED TO TRANSPLANTATION
- Conditioning regimen
- Graft-versus-host disease prophylaxis
- Infectious disease prophylaxis
- Liver complications
- Cardiac complications
- GRAFT REJECTION AND THALASSEMIA RECURRENCE
- Use of antithymocyte globulin
- Second transplant
- MIXED CHIMERIC STATE
- Transient mixed chimerism
- Persistent mixed chimerism
- EVALUATION FOLLOWING TRANSPLANTATION