在MASH引起的补偿性肝硬化中使用efruxifermin
Mazen Noureddin1,2, Mary E Rinella3, Naga P Chalasani4
1Houston Methodist Hospital, Houston.
The New England journal of medicine
|May 9, 2025
概括
在36周的MASH引起的补偿性肝硬化患者中,埃弗鲁西弗林没有显著降低纤维化. 96周的长期数据显示了潜在的益处,需要进一步调查MASH相关的肝病.
科学领域:
- 肝病学 肝病学是一种肝病学.
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 代谢功能障碍相关的脂肪肝炎 (MASH) 可能导致晚期肝纤维化,包括补偿性肝硬化 (4期).
- 作为纤维细胞生长因子21 (FGF21) 的类似物,Efruxifermin在MASH早期的纤维化减少方面表现有前途.
- 对于MASH诱导的补偿性肝硬化患者的efruxifermin,需要有效性和安全性数据.
研究的目的:
- 为了评估efruxifermin的疗效和安全性,在生体检查证实由MASH引起的补偿性肝硬化患者.
- 为了评估纤维化减少和36周和96周治疗后的MASH状态.
主要方法:
- 进行了一项2b期,随机,安慰剂控制,双盲试验.
- 患有MASH和补偿性肝硬化的患者接受皮下efruxifermin (28毫克或50毫克每天) 或安慰剂.
- 主要结局是至少有一个纤维化减少的阶段,在36周后MASH没有恶化.
主要成果:
- 在36周,在efruxifermin组和安慰剂组之间没有观察到纤维化减少的显著差异.
- 在96周,50毫克的efruxifermin组中,与安慰剂 (11%) 相比,更高比例的患者实现了纤维化减少 (29%).
- 胃肠道不良事件更频繁地发生在efruxifermin,大多是轻度至中度.
结论:
- 在36周的MASH相关补偿性肝硬化患者中,efruxifermin没有显示出显著的纤维化减少.
- 50毫克的剂量在96周后显示出潜在的益处,这表明可能有更长期的治疗效果.
- 需要进一步的研究来确认efruxifermin在这个患者群体中的疗效和安全性.
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