Department of Hematology, The First People's Hospital of Yunnan Province, The Affiliated Hospital of Kunming University of Science and Technology, Kunming, Yunnan, China.
Yunnan Province Clinical Research Center for Hematologic Disease, The First People's Hospital of Yunnan Province, Kunming, Yunnan, China.
Front Immunol. 2024 Aug 5;15:1403458. doi: 10.3389/fimmu.2024.1403458. eCollection 2024.
Genetic mutations in the β-globin gene lead to a decrease or removal of the β-globin chain, causing the build-up of unstable alpha-hemoglobin. This condition is referred to as beta-thalassemia (BT). The present treatment strategies primarily target the correction of defective erythropoiesis, with a particular emphasis on gene therapy and hematopoietic stem cell transplantation. However, the presence of inefficient erythropoiesis in BT bone marrow (BM) is likely to disturb the previously functioning BM microenvironment. This includes accumulation of various macromolecules, damage to hematopoietic function, destruction of bone cell production and damage to osteoblast(OBs), and so on. In addition, the changes of BT BM microenvironment may have a certain correlation with the occurrence of hematological malignancies. Correction of the microenvironment can be achieved through treatments such as iron chelation, antioxidants, hypoglycemia, and biologics. Hence, This review describes damage in the BT BM microenvironment and some potential remedies.
β-珠蛋白基因的基因突变导致β-珠蛋白链的减少或缺失,从而导致不稳定的α-血红蛋白的积累。这种情况被称为β-地中海贫血(BT)。目前的治疗策略主要针对纠正有缺陷的红细胞生成,特别强调基因治疗和造血干细胞移植。然而,BT 骨髓(BM)中低效的红细胞生成可能会扰乱之前功能正常的 BM 微环境。这包括各种大分子物质的积累、造血功能的损害、破骨细胞生成的破坏和成骨细胞(OBs)的损害等。此外,BT BM 微环境的变化可能与血液恶性肿瘤的发生有一定的相关性。微环境的纠正可以通过铁螯合、抗氧化剂、低血糖和生物制剂等治疗方法来实现。因此,本综述描述了 BT BM 微环境的损伤和一些潜在的治疗方法。