β-地中海贫血症:基于药物的治疗

Shrabonti Biswas, S. Smrity, Md. Shimul Bhuia, Fatema Akter Sonia, Mst. Asma Aktar, R. Chowdhury, Tawhida Islam, Muhammad Torequl Islam, Gabriel Gonçalves Alencar, C. Paulo, Ana Pavla Almeida Diniz Gurgel, H. Coutinho
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摘要

本综述旨在确定能消除或减少β(β)地中海贫血患者输血需求的药物的潜力。我们使用 PubMed Central、Google Scholar、PubMed 和 ScienceDirect 上从 1996 年至 2023 年 11 月发表的存档文章进行了全面的文献检索,以确定临床试验和研究。根据该综述,为了改善β地中海贫血症的治疗策略,已对多种药物进行了临床试验,其中包括卢帕特罗、索特罗、米他匹伐、依他沃匹伐、羟基脲、雷帕霉素、地西他滨、沙利度胺和槲皮素。在这些药物中,鲁帕他赛和索他他赛的疗效尤为显著,并已获得美国食品药品管理局(FDA)批准,可用于治疗β-地中海贫血患者。luspatercept 和 sotatercept 这两种药物的作用方式是刺激血红蛋白(Hb)的生成或增强其功能,从而减少对输血的依赖并提高整体生活质量。因此,luspatercept 和 sotatercept 等药物提供了一个机会,可显著减少 β 地中海贫血症患者输血的必要性,改善他们的生活水平和整体预后。然而,还需要更多的研究来评估这些药物的长期有效性和安全性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Beta-Thalassemia: A Pharmacological Drug-Based Treatment
This review was performed to determine the potential of drugs that can remove or decrease the requirements for blood transfusion among beta (β)-thalassemia patients. A comprehensive literature search was conducted to identify clinical trials and studies using PubMed Central, Google Scholar, PubMed, and ScienceDirect archived articles published from 1996 to November 2023. According to this review, clinical trials for a number of drugs, including luspatercept, sotatercept, mitapivat, etavopivat, hydroxyurea, rapamycin, decitabine, thalidomide, and quercetin, have been performed as part of efforts to improve the cure strategy for β-thalassemia. Of these drugs, luspatercept and sotatercept have exhibited particularly promising results and have been granted US Food and Drug Administration (FDA) approval for use in β-thalassemia patients. The mode of action for the drugs luspatercept and sotatercept involves the stimulation of hemoglobin (Hb) production or enhancement of its functionality, thereby decreasing reliance on blood transfusions and enhancing the overall quality of life. In this way, drugs like luspatercept and sotatercept present an opportunity to notably decrease the necessity for blood transfusions in β-thalassemia patients, improving their standard of living and overall prognosis. However, more research is needed to evaluate the effectiveness and safety of these drugs in the long run.
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