Mitapivat represents a significant advance in thalassemia management by targeting erythrocyte metabolism and providing the first FDA‐approved oral treatment for anemia in adults with α or β thalassemia.
Abstract
Thalassemia is an inherited hemoglobin disorder characterized by ineffective erythropoiesis, chronic anemia, and progressive multisystem complications that require lifelong management. Current treatment relies on regular red blood cell transfusions, iron chelation therapy, and supportive multidisciplinary care, while hematopoietic stem cell transplantation offers a curative option for selected patients. Although gene‐based therapies and novel pharmacological agents have expanded therapeutic options, their widespread implementation is limited by cost, accessibility, and long‐term safety considerations. This article summarizes contemporary management strategies for α and β thalassemia, with particular emphasis on the recent emergence of the FDA‐approved oral pyruvate kinase activator, mitapivat.
Several promising approaches targeting fetal hemoglobin induction, iron metabolism, and ineffective erythropoiesis have failed to demonstrate sufficient clinical benefit despite preclinical proof of concept, highlighting the complexity of therapeutic development in β-thalassemia.
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INTRODUCTION
Hemophilia A is an inherited bleeding disorder due to factor VIII (FVIII) deficiency and is characterized by recurrent bleeding and progressive joint damage. For the last two decades, treatment strategies have expanded from FVIII replacement therapy to include extended half-life products, non-factor therap...
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This review summarizes the recent advances in the pharmacological management of CKD-associated anemia, focusing on iron supplementation, ESAs, and HIF-PHIs.
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