Levy Maayan V, Ginzburg Yelena Z
The Tisch Cancer Institute, Division of Hematology and Medical Oncology, Tisch Cancer Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Adv Exp Med Biol. 2025;1480:145-161. doi: 10.1007/978-3-031-92033-2_11.
Erythropoiesis is a process by which red blood cells (RBCs) are produced in the adult bone marrow. It involves the differentiation of erythroid progenitor cells into mature RBCs, which transport oxygen from the lungs to all cells in the body. Erythropoiesis is a complex process with a nuanced crosstalk of regulation involving hormones, cytokines, and growth factors. In recent years, multiple sources of evidence have increased our understanding of the molecular mechanisms that coordinate erythropoiesis to enable the daily production of approximately 200 billion RBCs. These advances have shed light on the pathophysiology in a variety of diseases, i.e., iron-loading anemias, and are paving the way for novel therapeutic strategies in preclinical and clinical development to treat such dyserythropoietic disorders. This chapter elucidates our current understanding of iron-loading anemias in general and specifically in β-thalassemia and myelodysplastic syndrome (MDS), describes the current cutting-edge understanding in pathophysiology, and delineates what novel therapies are currently being developed to target these disorders.
红细胞生成是成年骨髓中产生红细胞（RBCs）的过程。它涉及红系祖细胞分化为成熟红细胞，成熟红细胞将氧气从肺部输送到身体的所有细胞。红细胞生成是一个复杂的过程，存在涉及激素、细胞因子和生长因子的细微调节串扰。近年来，多种证据来源增进了我们对协调红细胞生成以实现每日约2000亿个红细胞生成的分子机制的理解。这些进展揭示了各种疾病（即铁过载性贫血）的病理生理学，并为临床前和临床开发中治疗此类红细胞生成异常疾病的新型治疗策略铺平了道路。本章阐述了我们目前对一般铁过载性贫血的理解，特别是对β地中海贫血和骨髓增生异常综合征（MDS）的理解，描述了病理生理学方面当前的前沿认识，并描绘了目前正在开发的针对这些疾病的新型疗法。
