Mitapivat is the first in class oral allosteric activator of pyruvate kinase enzyme, leading to increased ATP production. Since red blood cells (RBC) rely on anaerobic metabolism, converting phosphoenolpyruvate to pyruvate in Embden– Meyerhof glycolytic pathway is the most important step for ATP production. Deficiency of ATP in patients with pyruvate kinase deficiency (PKD) leads to the destruction of RBCs. In hemoglobinopathies like thalassemia and sickle cell disease, increased stress and utilization leads to rapid depletion of ATP resources. Phase II DRIVE PK study was the first randomized controlled trial that showed benefits in adult patients without regular transfusion requirement in terms of a rise in hemoglobin ≥1.0 g/dl and improvement in other parameters of hemolysis even with a low 50 mg twice daily dose. Minor side effects like headache, insomnia, and nausea were reported. Subsequent adult studies like ACTIVATE III (non-transfusion-dependent) and ACTIVATE III – T (transfusion-dependent) in patients with PKD showed sustained hemoglobin response in 16/40 (40%) patients. It was tolerated well, and the side effect profile was similar to the previous study except for hypertriglyceridemia and hypertension in two patients. Phase I/II trials on patients with thalassemia and sickle cell anemia have also shown promising results in reducing transfusion burden and other disease-related co-morbidities, paving the way for further studies. Mitapivat appears to be a safe, well-tolerated, and effective drug for PKD and other RBC pathologies in adults. Results of ongoing pediatric studies in these settings are awaited to reveal its safety profile in children.

Ritika Khurana

Sangeeta Mudaliar

Pediatric Hematology Oncology Journal

The journal aims to promote high quality research and serve as a platform for dissemination of scientific information in pediatric hematology oncology with focus on research emanating from India and surrounding countries in the subcontinent, South Asia, West and East Asia, Africa, and serve as an alternative destination for Australia, Europe and the Americas to become a truly global and representative publication. The journal intends to be a one-stop resource for cutting edge research in all matters related to Pediatric Hematology and Oncology including    Hematology  Medical oncology  Surgical oncology  Radiation oncology  Imaging sciences  Hemato-pathology  Surgical pathology  Epidemiology.

The Pediatric Hematology Oncology Journal aims to promote high-quality research and serve as a platform for disseminating scientific information in the field of pediatric hematology and oncology. It focuses on research originating from India and surrounding countries in South Asia, West and East Asia, and Africa, while also serving as an alternative destination for researchers from Australia, Europe, and the Americas, striving to become a truly global and representative publication. The journal intends to be a one-stop resource for cutting-edge research in all matters related to pediatric hematology and oncology, including hematology, medical oncology, surgical oncology, radiation oncology, imaging sciences, hematopathology, surgical pathology, and epidemiology.

《Pediatric Hematology Oncology Journal》旨在促进高质量的研究，并为小儿血液肿瘤学领域提供一个传播科学信息的平台，特别关注来自印度及周边南亚、西亚、东亚、非洲的研究。同时，该期刊也希望成为澳大利亚、欧洲和美洲研究者的替代选择，成为一个真正全球化和具有代表性的出版物。期刊致力于成为小儿血液学和肿瘤学相关领域前沿研究的一站式资源，涵盖小儿血液学、医学肿瘤学、外科肿瘤学、放射肿瘤学、影像科学、血液病理学、外科病理学和流行病学等各个方面。

Mitapivat: New dawn in pyruvate kinase deficiency and beyond

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