Stem cell treatment cures thalassemia patients
Thalassemia, one of the most common genetic disorders, is considered to be a global problem. Several millions of the patients suffer from severe thalassemic diseases. Stem cell transplantation is currently the only curative therapy. Bone marrow transplantation offers a high probability of cure when performed in young children. There is a higher risk as the patient becomes older, especially the high incidence of graft rejection. Modified conditioning regimens have been developed to overcome graft rejection in patients with class III or full blown manifestations. The alternative use of stem cell from cord blood makes possible earlier transplant with better chance of cure, although the engraftment is slower compared to bone marrow transplantation. More experiences with regard to stem cell transplantation in adult patients, the use of stem cell transplantation from related donors as well as matched unrelated donors are necessary. Allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia major has been a cornerstone of the development of cell-based therapies and a matter of passionate debate during the ‘80s and early ‘90s.The aim of HSC transplantation in thalassemia is to replace the ineffective endogenous thalassemic erythropoiesis with an effective allogeneic substitute and to obtain a lasting, permanent, clinically effective correction of the hemolytic anemia and of the transfusion-associated pathologies, including iron overload. The large Pesaro experience demonstrated that this target has been achieved . Stem Cells, present in the bone marrow, give rise to red blood cells that produce Hemoglobin. In Thalassemia, it’s the defect in the stem cells that leads to deformed red blood cells that produce the abnormal hemoglobin and transfusion – dependent anemia. Stem Cell transplantation provides a thalassemia patient with healthy stem cells that will produce normal blood cells.
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