Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy.
Division of Hematology, University of Toronto, Toronto, ON, Canada.
Hematol Oncol Clin North Am. 2023 Apr;37(2):449-462. doi: 10.1016/j.hoc.2022.12.010.
Advances in understanding the underlying pathophysiology of β-thalassemia have enabled efforts toward the development of novel therapeutic modalities. These can be classified into three major categories based on their ability to target different features of the underlying disease pathophysiology: correction of the α/β globin chain imbalance, targeting ineffective erythropoiesis, and targeting iron dysregulation. This article provides an overview of these different emerging therapies that are currently in development for β-thalassemia.
对β-地中海贫血潜在病理生理学的认识的提高，使人们能够努力开发新的治疗方法。这些方法可以根据其靶向潜在疾病病理生理学不同特征的能力分为三大类：纠正α/β 球蛋白链失衡、靶向无效红细胞生成和靶向铁失调。本文概述了目前正在开发用于β-地中海贫血的这些不同的新兴疗法。
