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瑞美替隆:代谢功能障碍相关脂肪性肝病(MASLD)治疗的一项突破。

Resmetirom: A Breakthrough in the Treatment of Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD).

作者信息

Shakeel Laiba, Shaukat Ayesha, Akilimali Aymar

机构信息

Department of Internal Medicine Dow University of Health Sciences Karachi Pakistan.

Department of Research Medical Research Circle (MedReC) Goma Democratic Republic of the Congo.

出版信息

Health Sci Rep. 2025 Jun 18;8(6):e70920. doi: 10.1002/hsr2.70920. eCollection 2025 Jun.

Abstract

BACKGROUND AND AIMS

Metabolic dysfunction-associated steatotic liver disease (MASLD), previously referred to as non-alcoholic fatty liver disease (NAFLD), has become the most prevalent chronic liver disease worldwide. Its increasing incidence is closely linked with rising rates of obesity, Type 2 diabetes mellitus (T2DM), and metabolic syndrome. MASLD includes a spectrum of liver pathologies such as fibrosis, cirrhosis, and metabolic dysfunction-associated steatohepatitis (MASH). This review explores diagnostic advancements and therapeutic developments, focusing on Resmetirom, a selective thyroid hormone receptor-beta (THR-β) agonist, as a novel pharmacologic intervention.

METHODS

The review draws upon current literature, clinical guidelines, and data from pivotal randomized controlled trials to evaluate the diagnostic criteria, non-invasive assessment tools, lifestyle recommendations, and emerging pharmacotherapy for MASLD and MASH, focusing on Resmetirom.

RESULTS

Resmetirom has demonstrated significant efficacy in reducing hepatic fat content and improving markers of liver fibrosis in patients with noncirrhotic MASH and fibrosis stages F2-F3. Clinical trials showed improved liver histology, lipid profiles, and MASH resolution without fibrosis progression. Despite its promising profile, gastrointestinal side effects and gallstone-related complications have been observed. Long-term safety and outcomes related to cirrhosis or hepatocellular carcinoma prevention remain under investigation.

CONCLUSION

MASLD is a significant public health concern driven by metabolic dysfunction. While lifestyle changes remain central to management, Resmetirom offers a promising pharmacological option for patients with moderate to advanced MASH. Further long-term studies are needed to fully establish its safety, effectiveness, and role in preventing advanced liver disease.

摘要

背景与目的

代谢功能障碍相关脂肪性肝病(MASLD),以前称为非酒精性脂肪性肝病(NAFLD),已成为全球最普遍的慢性肝病。其发病率的上升与肥胖、2型糖尿病(T2DM)和代谢综合征发病率的上升密切相关。MASLD包括一系列肝脏病变,如纤维化、肝硬化和代谢功能障碍相关脂肪性肝炎(MASH)。本综述探讨了诊断进展和治疗进展,重点关注新型药物干预手段——选择性甲状腺激素受体β(THR-β)激动剂Resmetirom。

方法

本综述借鉴当前文献、临床指南以及关键随机对照试验的数据,以评估MASLD和MASH的诊断标准、非侵入性评估工具、生活方式建议和新兴药物治疗,重点关注Resmetirom。

结果

Resmetirom已证明在降低非肝硬化MASH和F2-F3纤维化阶段患者的肝脏脂肪含量以及改善肝纤维化标志物方面具有显著疗效。临床试验表明肝脏组织学、血脂谱得到改善,MASH消退且无纤维化进展。尽管其前景广阔,但已观察到胃肠道副作用和胆结石相关并发症。与肝硬化或肝细胞癌预防相关的长期安全性和结果仍在研究中。

结论

MASLD是由代谢功能障碍引发的重大公共卫生问题。虽然生活方式改变仍然是管理的核心,但Resmetirom为中度至重度MASH患者提供了一种有前景的药物选择。需要进一步的长期研究来充分确定其安全性、有效性以及在预防晚期肝病中的作用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a2f6/12174658/87a4f08aac77/HSR2-8-e70920-g001.jpg

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