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Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation

14 Mar 2025

Abstract excerpt

ABSTRACT: β-thalassemia (β-thal) is a worldwide hereditary red cell disorder characterized by severe chronic anemia. Recently, the pyruvate kinase (PK) activator mitapivat has been shown to improve anemia and ineffective erythropoiesis in a mouse model of β-thal and in patients with non-transfusion-dependent thalassemia. Here, we showed that in vitro CD34+-derived erythroblasts from patients with β-thal (codb039)...

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PMID: 40085946
DOI: 10.1182/bloodadvances.2024013591

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