Haema 2011; 2(3): 304-312
by Vasilis Ladis, Stelios Graphakos
Saint Sophia Children’s Hospital, Athens.
Abstract
Conventional therapy of Thalassaemia aims to maintain a “physiologic” haemoglobin level, to prevent iron accumulation and to establish normal development .Current therapeutic modalities include regular transfusions and intensive chelation, additionally with splenectomy, management of complications due to haemosidirosis and patients’ psychological supportive care. Effectiveness and safety of blood transfusions depends on careful monitoring of red blood units and selection of leucocyte-poor blood products. Splenectomy is indicated when transfusion requirements are excessive and attention should be given to the prevention of infections and thrombotic episodes in splenectomised patients. Haematopoietic stem cell transplantation (HSCT) remains the only curative approach for thalassaemic patients. The majority of the transplants have been performed from an HLA-matched sibling donor, although alternative donors have been used, but results are not satisfactory. Recent data indicate a probability of thalassaemia-free survival around 85%, rejection rate 10% and transplant relate mortal- ity between 5-15%. Mixed chimerism is a common phenomenon post HSCT for thalassaemia and it is of interest that patients presented with persistent mixed chimerism, are still able to maintain a normal haemoglobin level avoiding transfusions. “Ex-thalassaemics” must have a regular follow-up and early institution of iron removal. Best results are achieved in patients transplanted from an identical sibling donor and when HSCT is performed at a young age, early in the course of the disease.