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代谢功能障碍相关脂肪性肝病治疗的新兴疗法及实际应用

Emerging therapies and real-world application of metabolic dysfunction-associated steatotic liver disease treatment.

作者信息

Kim Hee Yeon, Rinella Mary E

机构信息

Department of Internal Medicine, Bucheon St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.

Division of Gastroenterology, Hepatology and Nutrition, University of Chicago Pritzker School of Medicine, Chicago, IL, USA.

出版信息

Clin Mol Hepatol. 2025 Jul;31(3):753-770. doi: 10.3350/cmh.2025.0083. Epub 2025 Apr 2.

Abstract

Metabolic dysfunction-associated steatotic liver disease, formerly referred to as non-alcoholic fatty liver disease, is the most common liver disease in Western countries and has emerged as the leading indication for liver transplantation. Metabolic dysfunction-associated steatohepatitis (MASH), a more advanced stage, carries a high risk of progression to liver fibrosis, cirrhosis, liver failure, and hepatocellular carcinoma. Until recently, lifestyle intervention remained the mainstay of MASH management, with no pharmacological treatments specifically approved. However, advances in understanding its pathophysiological mechanisms have fueled numerous clinical trials, culminating in the Food and Drug Administration's (FDA) approval of resmetirom as the first treatment for MASH in 2024. Additionally, many investigational drugs are nearing FDA approval or progressing through late-stage clinical trials. This review examines the current therapeutic landscape, highlights strategies for identifying patients suitable for liver-directed therapies in real-world settings, and discusses the challenges that remain.

摘要

代谢功能障碍相关脂肪性肝病,以前称为非酒精性脂肪性肝病,是西方国家最常见的肝脏疾病,已成为肝移植的主要指征。代谢功能障碍相关脂肪性肝炎(MASH)是更晚期阶段,具有进展为肝纤维化、肝硬化、肝衰竭和肝细胞癌的高风险。直到最近,生活方式干预仍是MASH管理的主要手段,尚无专门获批的药物治疗方法。然而,对其病理生理机制认识的进展推动了众多临床试验,最终美国食品药品监督管理局(FDA)于2024年批准resmetirom作为首个治疗MASH的药物。此外,许多研究性药物已接近FDA批准或正在进行后期临床试验。本综述审视了当前的治疗格局,强调了在现实环境中识别适合肝脏定向治疗患者的策略,并讨论了仍然存在的挑战。

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