Beta-thalassemia: is cure still a dream?
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Edizioni Minerva Medica
Abstract
β-thalassemia is a monogenic disorder characterized by decreased hemoglobin production, resulting in chronic anemia. There are several factors affecting the clinical presentation of patients with β-thalassemia, and several complications such as iron overload or ineffective erythropoiesis have been linked to this disease. Until nowadays, several conservative therapies namely blood transfusions, iron chelation, and the FDA-approved drug Luspatercept have been adopted alongside other debatable permanent cures. Other clinical trials are being conducted to develop better and safer management techniques for these patients. This review will discuss the different treatment strategies of β-thalassemia including novel therapies, besides all possible curative therapies that are being developed for this disease. © 2023 Edizioni Minerva Medica. All rights reserved.
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Anemia, Beta-thalassemia, Erythropoiesis inhibiting factors, Iron overload, Humans, Hemoglobin, Pyruvate kinase, Smad protein, Small interfering rna, Transforming growth factor beta, Transmembrane protease serine 2, Beta thalassemia, Blood clotting disorder, Blood transfusion, Erythropoiesis, Gene editing, Gene therapy, Hematopoietic stem cell transplantation, Human, Iron chelation, Pulmonary hypertension, Review, Splenectomy, Stem cell transplantation, Complication