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Reports liver risk and assessment needs in adults with thalassemia using Mitapivat.
Mitapivat (Aqvesme) has been approved to treat anemia in adults with α- or β-thalassemia. It is the first oral treatment for β-thalassemia and the first drug approved to treat α-thalassemia. Mitapivat's labeling carries a boxed warning that it increases the risk of liver damage and requires repeat laboratory tests to assess liver functioning.
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Diane S. Aschenbrenner (2026) studied this question.
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