Cure of Thalassemia

Hematopoietic Stem cell transplant (HSCT), commonly called Bone Marrow Transplantation (BMT) is the only curative therapy for thalassemia major and is well established curative therapy. HSCT should be offered to thalassemia patients at an early age, before complication due to iron overload have developed, if an HLA identical sibling is available. However older children, with inadequate chelation, liver enlargement, fibrosis, splenomegaly are at high risk of adverse outcomes and complications to this procedure.  A matched unrelated donor can be used provided that high compatibility criteria for both HLA class I and II loci are met.