遗传性性的临床试验的结果测量和生物标志物:一个范围审查
Sue-Faye Siow1,2, Dennis Yeow1,3,4,5,6, Laura I Rudaks1,2,4,5
1Sydney Medical School, University of Sydney, Camperdown 2050, Australia.
Genes
|September 28, 2023
概括
标准化结果测量和生物标志物对于遗传性性 (HSP) 临床试验至关重要. 本次审查强调了在HSP研究中需要核心措施,验证的生物标志物和患者报告的结果.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 生物标志物 生物标志物
背景情况:
- 遗传性性 (HSP) 是一种进展性神经疾病,影响下肢性.
- 目前,没有疾病修饰治疗存在HSP,需要强大的临床试验方法.
- 标准化和验证的结果措施对于推进HSP研究和治疗开发至关重要.
研究的目的:
- 进行对遗传性性的结果测量和生物标志物的范围审查.
- 为未来的HSP研究提供建议,并确定研究缺口.
- 为开发HSP有效的临床试验提供信息.
主要方法:
- 搜索了主要的科学数据库,包括Embase,Medline,Scopus,科学网络和Cochrane中央.
- 包括70项符合预先定义的纳入标准的研究.
- 确定并分析了83种不同的结果指标和各种生物标志物.
主要成果:
- 性麻评分尺度 (SPRS) 是最常用的测量方法,其次是修改后的阿什沃思尺度和MRI.
- 患者报告的结果措施 (PROMs) 在评估治疗疗效时未得到充分利用 (28%的干预研究).
- 扩散张力成像,步态分析和神经纤维光链显示出与疾病严重程度相关的生物标志物具有前途.
结论:
- 在HSP的结果指标的应用中存在显著的变化和不一致.
- 在HSP临床试验中,有必要制定一套标准化的核心结果指标.
- 建议对有前途的生物标志物进行进一步验证,并将PROMs常规纳入未来的HSP研究.
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