Title:Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with
Pyruvate Kinase Deficiency
Volume: 31
Issue: 6
关键词:
溶血性贫血,丙酮酸激酶缺乏症,米他品,镰状细胞病,地中海贫血,红细胞,血红蛋白。
摘要: Mitapivat is an orally bioavailable small molecule allosteric activator of pyruvate
kinase. It was approved by the US FDA on February 17, 2022, and the European
Union in November 2022 for the treatment of hemolytic anemia in adult patients with pyruvate
kinase deficiency. In this short perspective, physicochemical properties, synthesis,
dosage and administration, mechanism of action, pharmacodynamics, pharmacokinetics,
drug interactions, and adverse reactions of mitapivat are described.