在加密球菌脑膜炎试验中对等级复合终点的结果排名 (DOOR) 和获胜率的可取性评估
Biyue Dai1, Nicole Engen1, Caleb P Skipper2
1Division of Biostatistics and Health Data Science, School of Public Health, University of Minnesota, Minneapolis, Minnesota, USA.
概括
层次复合终点通过包括死亡率和不良事件来提高抗真菌试验的相关性. 这些方法增强了对加密球菌脑膜炎治疗的临床试验分析.
科学领域:
- 临床试验 临床试验
- 传染性疾病 传染性疾病
- 生物统计学 生物统计学
背景情况:
- 传统的抗真菌试验通常使用全因死亡率作为主要终点.
- 这种方法可能无法完全捕捉关键的治疗结果,如不良事件或耐受性.
研究的目的:
- 在加密球菌脑膜炎临床试验中评估分层复合终点的实用性.
- 将患者的死亡率和安全优先考虑在试验结果评估中.
主要方法:
- 一项涉及34名专家的Delphi调查确定了复合材料终端构建的关键优先事项.
- 分析了三项随机临床试验 (AMBITION-cm,ENACT,ASTRO-CM) 使用获胜率和结果排名的可取性 (DOOR).
- 计算更好的DOOR概率 (BDP),以量化研究治疗更理想的结果的可能性.
主要成果:
- 德尔菲调查显示,对死亡率和严重不良事件的优先考虑很强.
- 在AMBITION-cm中,单剂量脂质体安波特里辛B显示出优越性 (BDP55%,胜率1.23).
- 在ENACT中的口服安福特里辛B和ASTRO-CM中的辅助塞特拉林在使用这些复合终点时没有显示出统计学上显著的益处.
结论:
- 阶层复合终点是可行的,并提高了加密球菌脑膜炎试验的临床相关性,特别是在非劣势性研究中.
- DOOR和Win Ratio提供互补的统计见解,DOOR提供绝对等级,Win Ratio估计相对治疗效应.
- 这些先进的终点更好地反映了治疗的疗效,安全性和耐受性,而不是传统的仅死亡率的措施.
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