Current and Future Treatments of Iron Overload in Thalassemia Patients

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Iron overload is a major complication among thalassemia patients. In thesepatients, ineffective erythropoiesis decreases hepcidin production resulting in irondysregulation, which leads to a number of serious complications. Damage to organssusceptible to iron overload could be prevented by effective iron chelation. Despite theefficacy of iron chelators, limitations to their use are that they are only used after thepatients have suffered from iron overload, and they have also been associated with anumber of side effects. New therapeutic strategies for the treatment of thalassemia havefocused on addressing the pathophysiology of the disease. Drugs currently beingdeveloped to improve ineffective erythropoiesis are aimed at increasing hemoglobinlevels and subsequently decreasing iron absorption. The new therapeutic drugs in thisclass include pegylated erythropoietin, JAK 2 inhibitors, and TGF-β activin receptortraps (sotatercept and luspatercept). Luspatercept is currently recognized as the mostpromising drug in this class and has completed phase III of trials. With the aim ofimproving iron dysregulation, these new therapeutic strategies focus on preventing theabsorption of iron in the gastrointestinal tract. These therapies involve hepcidinagonists and specific derivatives, such as LJPC-401 and Rusfertide (formerly PTG-300), certain ferroportin inhibitors, such as Vamifeport (formerly VIT-2763) andtransmembrane protease serine 6 (TMPRSS6) antisense oligonucleotides. Although thetherapeutic potential of these new treatments in thalassemia patients is promising,ongoing clinical trials are needed. Importantly, these new treatment strategies mayprovide a new, more effective paradigm of treatment in thalassemia patients.