Expert opinion on pharmacotherapy · 2026
Evaluating mitapivat for the treatment of alpha or beta thalassemia.
СтатьяОбзорНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Kattamis Antonis, Bistas Konstantinos, Mitros Vangelis, Agiomavriti Stefanopoulou Efthalia Faidra, Delaporta Polyxeni
Аннотация
INTRODUCTION: Thalassemia is a group of diverse genetic disorders with worldwide distribution that affects hemoglobin synthesis. Until recently, the therapeutic approach to thalassemia was symptomatic, relying on red blood cell transfusions, treatment of comorbidities and of disease-related complications. However, novel therapeutic agents have recently been developed and are gradually being integrated into routine clinical practice.
AREAS COVERED: One of the most promising agents is mitapivat (AG348), an oral pyruvate kinase activator that enhances the erythrocytic adenosine triphosphate (ATP) production. After series of preclinical and clinical studies, mitapivat has been suggested to be a safe and effective disease modifier for thalassemia. Large double blind randomized clinical trials have indicated that mitapivat may increase baseline hemoglobin levels, reduce transfusion burden, control ineffective erythropoiesis and hemolysis and improve quality of life.
EXPERT OPINION: Mitapivat presents as a potential game-changer in the management of patients with both α- and β-thalassemia, regardless of transfusion dependency. However, further post-marketing evidence is required in order to evaluate mitapivat profile under real‑world conditions and routine clinical practice.
AREAS COVERED: One of the most promising agents is mitapivat (AG348), an oral pyruvate kinase activator that enhances the erythrocytic adenosine triphosphate (ATP) production. After series of preclinical and clinical studies, mitapivat has been suggested to be a safe and effective disease modifier for thalassemia. Large double blind randomized clinical trials have indicated that mitapivat may increase baseline hemoglobin levels, reduce transfusion burden, control ineffective erythropoiesis and hemolysis and improve quality of life.
EXPERT OPINION: Mitapivat presents as a potential game-changer in the management of patients with both α- and β-thalassemia, regardless of transfusion dependency. However, further post-marketing evidence is required in order to evaluate mitapivat profile under real‑world conditions and routine clinical practice.