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基于FVC的有效性终点的贝叶斯推断,从成年人到接受Nintedanib的儿科患者
Sonja Hartmann1, Anna Chan Kwong2, Jakob Ribbing2
1Translational Medicine and Clinical Pharmacology, Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, Connecticut, USA.
CPT: pharmacometrics & systems pharmacology
|December 9, 2025
概括
丁丁达尼布在儿童中有效治疗渐进性纤维化间歇性肺病,其安全性和疗效与成人相似. 这项研究支持在患有这些罕见肺部疾病的儿科患者中使用它.
科学领域:
- 药理学和毒理学 药理学和毒理学
- 肺部病理学 肺部病理学
- 儿科医学 儿科医学
背景情况:
- 尼丁达尼布已批准用于患有异常性肺纤维化 (IPF) 和其他渐进性纤维化间歇性肺病 (PF-ILD) 的成年人.
- 在PF-ILD中使用宁泰达尼布的儿科数据有限,因此需要从成人研究中推断.
研究的目的:
- 评估宁泰达尼布在患有渐进性纤维化ILD的儿科患者的药理动力学 (PK),疗效和安全性.
- 为了支持6至17岁儿童对宁泰达尼布的剂量选择和益处风险评估.
主要方法:
- 进行了一项儿科第3期试验 (InPedILD) 和其扩展 (InPedILD-ON).
- 从成人研究中获得的人口PK和暴露-反应 (ER) 模型,使用贝叶斯方法对儿科患者进行了部分推断.
- 分析了PK和疗效终点 (FVC %预测,FVC Z-score) 的结果.
主要成果:
- 基于体重的小儿用剂量达到与有效的成人剂量相比的暴露.
- 暴露-反应模型表明,与成年人相比,儿科患者的FVC衰减率相似,且较慢.
- 估计的EC50和Emax值在儿科和成人群体之间是可比的.
结论:
- 从成年人数据的部分推断支持儿科宁泰达尼布剂量方案的有效性和安全性.
- 丁丁达尼布在患有渐进性纤维化ILD的儿童和成人中对FVC表现出类似的有益治疗效果.
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