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用于非酒精性脂肪性肝炎的雷斯美洛:对一个新型治疗前沿领域的全面综述

Resmetirom for MASH: A Comprehensive Review of a Novel Therapeutic Frontier.

作者信息

Tiwari Angad, Sharma Ashish, Kumar Harendra, Gupta Varnika, Deshpande Vishal, Mupparaju Jaya Sai, Mishra Tanisha, Bharadwaj Hareesha Rishab, Dahiya Dushyant Singh, Jain Varun

机构信息

Department of Internal Medicine, Maharani Laxmi Bai Medical College, Jhansi 284001, Uttar Pradesh, India.

Department of Internal Medicine, Yale New Haven Hospital, New Haven, CT 06504, USA.

出版信息

Biomedicines. 2025 Aug 26;13(9):2079. doi: 10.3390/biomedicines13092079.

Abstract

Metabolic dysfunction-associated steatohepatitis (MASH) is a progressive liver disease linked to type 2 diabetes (T2D), obesity, and dyslipidaemia, which are all parts of the metabolic syndrome. In 2024, for non-cirrhotic MASH with mild to advanced fibrosis, resmetirom, a selective thyroid hormone receptor-β agonist, became the first FDA-approved treatment for this condition. By increasing β-oxidation and lipid metabolism, it minimises systemic thyroid or cardiac effects while reducing hepatic fat, inflammation, and fibrosis. Resmetirom is being developed for use in combination with lifestyle interventions, such as diet and exercise, to maximize patient benefit. Nevertheless, the lack of congruence between clinical trial populations and real-world payer criteria underscores access obstacles that necessitate policy reform. The successful delivery of screening programs depends on the education of providers from various disciplines and the establishment of uniform screening standards. Future studies should investigate the clinical application of resmetirom in combination with agents that may provide additional benefits, such as GLP-1 receptor agonists, SGLT2 inhibitors, and statins. These results are significant in light of recent long-term safety monitoring of these agents, particularly regarding the thyroid axis. Ensuring equitable uptake will be crucial, as it involves defining fair access through payer endpoints, conducting cost-effectiveness analysis, and considering patient-reported outcomes. Resmetirom represents a breakthrough in MASH management, offering potential metabolic benefits in conjunction with comprehensive clinical and lifestyle approaches.

摘要

代谢功能障碍相关脂肪性肝炎(MASH)是一种与2型糖尿病(T2D)、肥胖和血脂异常相关的进行性肝病,这些都是代谢综合征的组成部分。2024年,对于伴有轻度至重度纤维化的非肝硬化MASH,选择性甲状腺激素受体-β激动剂瑞美替昂成为首个获美国食品药品监督管理局(FDA)批准用于该病症的治疗药物。通过增加β-氧化和脂质代谢,它在减少肝脏脂肪、炎症和纤维化的同时,将全身甲状腺或心脏影响降至最低。瑞美替昂正在研发中,用于与饮食和运动等生活方式干预措施联合使用,以最大化患者获益。然而,临床试验人群与现实世界支付方标准之间缺乏一致性,凸显了需要政策改革的获取障碍。筛查项目的成功实施取决于对各学科医疗服务提供者的教育以及统一筛查标准的建立。未来的研究应调查瑞美替昂与可能提供额外益处的药物(如胰高血糖素样肽-1受体激动剂、钠-葡萄糖协同转运蛋白2抑制剂和他汀类药物)联合使用的临床应用。鉴于近期对这些药物的长期安全性监测结果,尤其是关于甲状腺轴的监测结果,这些结果意义重大。确保公平获取至关重要,因为这涉及通过支付方终点定义公平获取、进行成本效益分析以及考虑患者报告的结果。瑞美替昂代表了MASH管理方面的一项突破,结合全面的临床和生活方式方法提供了潜在的代谢益处。

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