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Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Until recently, treatment had been limited to supportive management including red blood cell transfusions, splenectomy, and management of chronic disease complications such as iron overload and decreased bone mineral density. We discuss preclinical data and phase 1, 2, and 3 clinical studies evaluating mitapivat for adult patients with hemolytic anemia secondary to PKD. Mitapivat has been shown to offer early and durable improvement in hemoglobin with reduction in transfusion burden, and preliminary data suggest it can induce a negative iron balance in many patients without the use of dedicated iron chelators. Mitapivat is a first-in-class allosteric activator of pyruvate kinase and the first FDA-approved disease directed therapy for PKD. It has a favorable safety profile and clear clinical efficacy. Given the considerable genetic heterogeneity of PKD and the rapid effect on improving hemoglobin and reducing hemolysis, a therapeutic trial of mitapivat should be considered in all patients with PKD who are not homozygous for the  PKLR  R479H mutation. Further investigations are needed regarding long-term safety and efficacy profiles and whether long-term PKD-associated complications can be reduced or even reversed.

An evaluation of mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency