Kaya Eda, Yilmaz Yusuf, Alkhouri Naim
Department of Medicine, Knappschaftskrankenhaus Bochum, Ruhr University, Bochum, Germany.
The Global NASH Council, Washington, DC.
J Clin Gastroenterol. 2025;59(5):412-419. doi: 10.1097/MCG.0000000000002150. Epub 2025 Feb 13.
The recent conditional approval by the Food and Drug Administration of resmetirom for treating metabolic dysfunction-associated steatohepatitis (MASH) with significant or advanced fibrosis represents a pivotal milestone in the history of metabolic dysfunction-associated steatotic liver disease (MASLD) treatment. As the first liver-directed pharmacological therapy option for MASLD, resmetirom offers a novel approach that specifically targets liver pathology, marking a transformative step forward in managing this widespread and challenging condition. For initiating therapy with resmetirom, a liver biopsy is not required. Consequently, accurately excluding patients with less severe liver histology or cirrhosis using noninvasive tests (NITs) is essential. In addition, monitoring the therapy response should be conducted using NITs. Given the recent approval, our current clinical understanding of resmetirom is primarily informed by phase 3 clinical trials. The long-term effects of the drug should be evaluated in further studies by encouraging the use of the drug in eligible patients. This review highlights key aspects of clinical resmetirom use, including identifying the target population, monitoring therapeutic response, determining appropriate discontinuation criteria, and strategies to prevent unnecessary treatment interruptions.
美国食品药品监督管理局(Food and Drug Administration)最近有条件批准了resmetirom用于治疗伴有显著或晚期纤维化的代谢功能障碍相关脂肪性肝炎(MASH),这是代谢功能障碍相关脂肪性肝病(MASLD)治疗史上的一个关键里程碑。作为MASLD的首个肝脏靶向药物治疗选择,resmetirom提供了一种专门针对肝脏病理的新方法,标志着在管理这种广泛且具有挑战性的疾病方面向前迈出了变革性的一步。对于开始使用resmetirom进行治疗,不需要进行肝活检。因此,使用非侵入性检测(NITs)准确排除肝脏组织学较轻或无肝硬化的患者至关重要。此外,应使用NITs监测治疗反应。鉴于最近的批准,我们目前对resmetirom的临床认识主要基于3期临床试验。应鼓励在符合条件的患者中使用该药物,通过进一步研究评估该药物的长期效果。本综述重点介绍了resmetirom临床应用的关键方面,包括确定目标人群、监测治疗反应、确定合适的停药标准以及防止不必要治疗中断的策略。