在儿科低级结质瘤临床试验中考虑功能性结果作为有效性终点:FDA教育研讨会
Jason Fangusaro1, Robert A Avery2, Michael J Fisher2
1Children's Hospital of Atlanta, Emory University and the Aflac Cancer Center, Atlanta, Georgia.
概括
本次研讨会探讨了使用视觉和运动技能等功能性结果作为儿科低度质瘤 (pLGG) 临床试验中的疗效终点. 讨论了将这些评估纳入未来药物开发的关键考虑因素.
科学领域:
- 儿科神经瘤学 儿童神经瘤学
- 临床试验的设计
- 生物统计学 生物统计学
背景情况:
- 儿科低度质瘤 (pLGG) 是儿科神经瘤学的一个重大问题.
- 目前对PLGG的临床试验主要集中在瘤反应上,可能会忽视患者的长期福祉.
- 功能性结果越来越被认为是评估儿科患者治疗疗效的关键.
研究的目的:
- 讨论在儿科低度质瘤 (pLGG) 临床试验中将功能性结果作为疗效终点的潜力.
- 在选择,实施和评估功能性结果评估时识别挑战和机会.
- 概述将这些终点纳入未来临床试验设计和药物开发的关键考虑因素.
主要方法:
- 这项研究是基于FDA瘤学卓越中心主办的教育研讨会的讨论.
- 参与者包括患者倡导者,监管机构 (FDA,EMA) 和儿科神经瘤学国际专家.
- 关键主题包括视觉敏,运动功能和神经认知性能作为潜在的终点.
主要成果:
- 在PLGG试验中,有必要考虑功能性结果,而不仅仅是传统的瘤反应指标.
- 在对功能领域临床结果评估的选择,实施和评估进行标准化方面存在挑战.
- 机会在于完善试验设计,以更好地捕捉治疗对患者生活质量和长期功能的影响.
结论:
- 功能性结果,包括视觉,运动和神经认知评估,在pLGG临床试验中作为关键的疗效终点具有前景.
- 需要进一步的研究和达成共识,以克服使用这些终点的挑战.
- 将功能性结果整合到试验设计中,对于推动pLGG新疗法开发至关重要.
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