Current and Future Treatments of Iron Overload in Thalassemia Patients

Iron overload is a major complication among thalassemia patients. In these

patients, ineffective erythropoiesis decreases hepcidin production resulting in iron

dysregulation, which leads to a number of serious complications. Damage to organs

susceptible to iron overload could be prevented by effective iron chelation. Despite the

efficacy of iron chelators, limitations to their use are that they are only used after the

patients have suffered from iron overload, and they have also been associated with a

number of side effects. New therapeutic strategies for the treatment of thalassemia have

focused on addressing the pathophysiology of the disease. Drugs currently being

developed to improve ineffective erythropoiesis are aimed at increasing hemoglobin

levels and subsequently decreasing iron absorption. The new therapeutic drugs in this

class include pegylated erythropoietin, JAK 2 inhibitors, and TGF-β activin receptor

traps (sotatercept and luspatercept). Luspatercept is currently recognized as the most

promising drug in this class and has completed phase III of trials. With the aim of

improving iron dysregulation, these new therapeutic strategies focus on preventing the

absorption of iron in the gastrointestinal tract. These therapies involve hepcidin

agonists and specific derivatives, such as LJPC-401 and Rusfertide (formerly PTG-

300), certain ferroportin inhibitors, such as Vamifeport (formerly VIT-2763) and

transmembrane protease serine 6 (TMPRSS6) antisense oligonucleotides. Although the

therapeutic potential of these new treatments in thalassemia patients is promising,

ongoing clinical trials are needed. Importantly, these new treatment strategies may

provide a new, more effective paradigm of treatment in thalassemia patients.

Keywords: Activin receptor, Antisense oligonucleotides, Ferritin, Ferroportin, Ferroportin inhibitor, Hepcidin, Hepcidin agonist, Ineffective erythropoiesis, Iron overload, Iron chelator, JAK-2 inhibitor, Luspatercept, Pegylated erythropoietin, Rusfertide, Sotatercept, Thalassemia, Thalassemia complication, TMPRSS6, TGF-β, Vamifeport.

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DOI https://doi.org/10.2174/9789815039535122050005	Print ISSN 2467-9585
Publisher Name Bentham Science Publisher	Online ISSN 2352-3239

Frontiers in Clinical Drug Research: Hematology