Department of Pharmacy, Uppsala University, Uppsala, Sweden.
Bayer AG, Wuppertal, Germany.
CPT Pharmacometrics Syst Pharmacol. 2024 May;13(5):880-890. doi: 10.1002/psp4.13125. Epub 2024 Mar 12.
Obstructive sleep apnea (OSA) is a sleep disorder which is linked to many health risks. The gold standard to evaluate OSA in clinical trials is the Apnea-Hypopnea Index (AHI). However, it is time-consuming, costly, and disregards aspects such as quality of life. Therefore, it is of interest to use patient-reported outcomes like the Epworth Sleepiness Scale (ESS), which measures daytime sleepiness, as surrogate end points. We investigate the link between AHI and ESS, via item response theory (IRT) modeling. Through the developed IRT model it was identified that AHI and ESS are not correlated to any high degree and probably not measuring the same sleepiness construct. No covariate relationships of clinical relevance were found. This suggests that ESS is a poor choice as an end point for clinical development if treatment is targeted at improving AHI, and especially so in a mild OSA patient group.
阻塞性睡眠呼吸暂停(OSA)是一种与许多健康风险相关的睡眠障碍。评估临床试验中 OSA 的金标准是呼吸暂停-低通气指数(AHI)。然而,它既耗时又昂贵,并且忽略了生活质量等方面。因此,使用患者报告的结果,如嗜睡量表(ESS),来作为替代终点,这是很有意义的,ESS 可以衡量白天的嗜睡程度。我们通过项目反应理论(IRT)模型来研究 AHI 和 ESS 之间的联系。通过开发的 IRT 模型,确定 AHI 和 ESS 没有高度相关,可能没有测量相同的嗜睡结构。没有发现临床相关的协变量关系。这表明,如果治疗的目标是改善 AHI,ESS 作为临床开发的终点选择是不理想的,尤其是在轻度 OSA 患者群体中更是如此。