Exploring a Role for Mitapivat in Children with Pyruvate Kinase Deficiency

European Medical Journal Hematology

article

Rachael Grace¹ 

[1] Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School

DOI  :  10.33590/emjhematol/10057650

来源: European Medical Journal

【 摘 要 】

Three posters presented at the European Hematology Association (EHA)2022 Hybrid Congress focused on the role of mitapivat in the treatmentof pyruvate kinase (PK) deficiency, a rare inherited chronic haemolytic anaemia.1-3Mitapivat is a first-in-class, oral, allosteric activator of the red blood cell (RBC) PKenzyme, and treats the underlying pathophysiology of PK deficiency.1-3 Results froma long-term extension study in adults have confirmed the consistency and durabilityof treatment response achieved with mitapivat, providing the basis for its ongoingevaluation in the treatment of paediatric patients with PK deficiency.1 Currently,there are no approved pharmacotherapies that target the underlying cause ofhaemolytic anaemia in children with PK deficiency. To address this global unmetneed, recruitment is currently underway into two international Phase III paediatricstudies (ACTIVATE-KidsT [NCT05144256]4 and ACTIVATE-Kids [NCT05175105]5),which will evaluate efficacy and safety of mitapivat treatment in children with PKdeficiency who are regularly transfused and not regularly transfused, respectively.

【 授权许可】

CC BY-NC