A literature review published in De Gruyter Open Access concluded that ebselen is a promising adjuvant therapy in treating patients with iron-overloaded thalassemia.

Prior studies indicated that ebselen, an imitator glutathione peroxidase, can alleviate cardiac dysfunction in iron-overloaded situations. The authors of the study conducted a systematic review on various academic search engines to further uncover the role of ebselen in relieving cardiomyopathy associated with iron overload in patients with thalassemia. 

Out of an initial finding of 178 studies, a final 3 were included in this review. The researchers determined that ebselen primarily preserves cardiac function in iron-overloaded conditions by binding to intracellular iron and reducing the synthesis of reactive oxygen species, which is made possible by its antioxidant and anti-inflammatory properties. Additionally, co-administration of ebselen and desferrioxamine can improve cardiac function in mice with thalassemia with an iron burden by decreasing cardiac hemosiderosis, cardiac malondialdehyde, and plasma non-transferrin bound iron.


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Trials prescribing ebselen at 200 mg, 400 mg, and 600 mg twice daily in individuals without thalassemia found it to be safe and well-tolerated. There were 2 trials which found that the most common complication in taking ebselen was drowsiness (between 14% to 25%). Ebselen prescribed at a dose of 600 mg twice daily in a trial with manic/hypomanic patients found upper respiratory tract infection (13%) to be the most common adverse event, followed by insomnia (8%), pruritus (6%), rash (5%), and frequent urination (4%).

“Intriguingly, the properties of ebselen…make it an exciting and promising possibility for adjuvant therapy in patients with thalassemia alongside the standard treatment with iron chelators, particularly in severe cases with cardiomyopathy,” the authors of the study concluded. “However, clinical trials recruiting human subjects need to be carried out ahead of definitive conclusions depicting its effectiveness and safety.”

Reference

Ghazaiean M, Aliasgharian A, Karami H, Darvishi-Khezri H. Ebselen: a promising therapy protecting cardiomyocytes from excess iron in iron-overloaded thalassemia patientsOpen Med (Wars). 2023;18(1):20230733. doi:10.1515/med-2023-0733