Evidera, Ivry-sur-Seine, France.
George Washington University School of Medicine and Health Sciences, Washington, D.C., USA.
J Patient Rep Outcomes. 2024 Jul 15;8(1):73. doi: 10.1186/s41687-024-00751-7.
Patient-focused approaches to capturing day-to-day variability in sleep disturbance are needed to properly evaluate the sleep benefits of new treatments. Such approaches rely on patient-reported outcome (PRO) measures validated in the target patient population.
Using atopic dermatitis (AD) as an example of a disease in which sleep is commonly disturbed, we developed a strategy for measuring sleep disturbance in AD trials. In developing this strategy, we conducted a targeted literature review and held concept elicitation interviews with adolescents and adults with AD. We subsequently identified potentially suitable PRO measures and cognitively debriefed them. Finally, we evaluated their psychometric properties using data from phase 2b (NCT03100344) and phase 3 (NCT03985943 and NCT03989349) clinical trials.
The literature review confirmed that sleep disturbance is a key impact of AD but failed to identify validated PRO measures for assessing fluctuations in sleep disturbance. Subsequent concept elicitation interviews confirmed the multidimensional nature of sleep disturbance in AD and supported use of a single-item measure to assess overall sleep disturbance severity, complemented by a diary to capture individual components of sleep disturbance. The single-item sleep disturbance numerical rating scale (SD NRS) and multi-item Subject Sleep Diary (SSD)-an AD-adapted version of the Consensus Sleep Diary-were identified as potentially suitable PRO measures. Cognitive debriefing of the SD NRS and SSD demonstrated their content validity and their understandability to patients. Psychometric analyses based on AD trial data showed that the SD NRS is a well-defined, reliable, and fit-for-purpose measure of sleep disturbance in adults with AD. Furthermore, the SD NRS correlated with many SSD sleep parameters, suggesting that most concepts from the SSD can be covered using the SD NRS.
Using these findings, we developed an approach for measuring sleep disturbance in AD trials. Subject to further research, the same approach could also be applied to future trials of other skin diseases where itch causes sleep disturbance.
需要以患者为中心的方法来捕捉睡眠障碍的日常变化，以便正确评估新治疗方法对睡眠的益处。此类方法依赖于在目标患者人群中经过验证的患者报告结局（PRO）测量。
以特应性皮炎（AD）为例，这是一种常见的睡眠障碍疾病，我们制定了一种在 AD 试验中测量睡眠障碍的策略。在制定该策略时，我们进行了针对性的文献回顾，并与 AD 青少年和成年人进行了概念启发访谈。我们随后确定了潜在合适的 PRO 测量方法，并对其进行了认知性审核。最后，我们使用来自 2b 期（NCT03100344）和 3 期（NCT03985943 和 NCT03989349）临床试验的数据评估了它们的心理测量特性。
文献回顾证实，睡眠障碍是 AD 的关键影响因素，但未能确定用于评估睡眠障碍波动的经过验证的 PRO 测量方法。随后的概念启发访谈证实了 AD 中睡眠障碍的多维性质，并支持使用单一项目测量来评估整体睡眠障碍严重程度，辅以日记记录睡眠障碍的各个组成部分。单一项目睡眠障碍数字评定量表（SD NRS）和多项目主观睡眠日记（SSD-共识睡眠日记的 AD 适应版）被确定为潜在合适的 PRO 测量方法。SD NRS 和 SSD 的认知审核证明了它们的内容有效性和患者的理解性。基于 AD 试验数据的心理测量分析表明，SD NRS 是一种定义明确、可靠且适合于 AD 成人睡眠障碍的测量方法。此外，SD NRS 与 SSD 的许多睡眠参数相关，这表明 SSD 的大多数概念都可以用 SD NRS 来涵盖。
使用这些发现，我们开发了一种在 AD 试验中测量睡眠障碍的方法。在进一步研究的基础上，相同的方法也可以应用于其他因瘙痒引起睡眠障碍的皮肤病的未来试验。
