Faculty of Medicine and Health, University of Sydney, Camperdown, NSW, Australia.
Sleep Theme at Charles Perkins Centre, University of Sydney, Camperdown, NSW, Australia.
Sleep. 2022 Oct 10;45(10). doi: 10.1093/sleep/zsac156.
To systematically determine subjective and objective outcome measures used to measure the efficacy of narcolepsy interventions in randomized controlled trials (RCTs) in adults and children and assess psychometric properties of patient-reported outcome measures (PROMs) used.
We searched bibliographical databases and clinical trial registries for narcolepsy RCTs and extracted objective and subjective outcome measures. If PROMs were used, we searched for psychometric studies conducted in a narcolepsy population using bibliographical databases and appraised using Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) guidelines.
In total, 80 different outcome measures were used across 100 RCTs. Epworth Sleepiness Scale (ESS) (n = 49) and Maintenance of Wakefulness Test (n = 47) were the most frequently used outcome measures. We found 19 validation studies of 10 PROMs in narcolepsy populations. There was limited evidence for validity or responsiveness of the ESS; yet sufficient reliability (pooled ICC: 0.81-0.87). Narcolepsy Severity Scale (NSS) had sufficient reliability (pooled ICC: 0.71-0.92) and both adult and pediatric versions had sufficient discriminant validity (treated/untreated). Content validity was only evaluated in pediatric populations for ESS-CHAD and NSS-P and rated inconclusive. Quality of evidence of the psychometric studies for all scales ranged from very low to low.
Although recognized by regulatory bodies and widely used as primary outcome measures in trials, there is surprisingly little evidence for the validity, reliability, and responsiveness of PROMs frequently used to assess treatment efficacy in narcolepsy. The field needs to establish patient-centered minimal clinically important differences for the PROMs used in these trials.
系统地确定用于测量成人和儿童发作性睡病干预措施疗效的主观和客观结局测量指标，并评估用于测量的患者报告结局测量指标（PROM）的心理测量学特性。
我们搜索了发作性睡病随机对照试验（RCT）的文献数据库和临床试验登记处，并提取了客观和主观的结局测量指标。如果使用了 PROM，我们还会搜索发作性睡病患者群体中使用的心理测量学研究，使用共识基础的健康测量仪器选择标准（COSMIN）指南进行评估。
总共在 100 项 RCT 中使用了 80 种不同的结局测量指标。Epworth 嗜睡量表（ESS）（n = 49）和维持清醒试验（MWT）（n = 47）是最常使用的结局测量指标。我们发现 10 种 PROM 在发作性睡病患者群体中有 19 项验证研究。ESS 仅有有限的有效性或反应性证据；但具有足够的可靠性（汇总 ICC：0.81-0.87）。发作性睡病严重程度量表（NSS）具有足够的可靠性（汇总 ICC：0.71-0.92），成人和儿童版本均具有足够的区分效度（治疗/未治疗）。仅对 ESS-CHAD 和 NSS-P 进行了儿童群体的内容有效性评估，评定结果不明确。所有量表的心理测量学研究的证据质量从极低到低不等。
尽管监管机构认可并广泛用作试验的主要结局指标，但用于评估发作性睡病治疗效果的 PROM 的有效性、可靠性和反应性的证据很少。该领域需要为这些试验中使用的 PROM 建立以患者为中心的最小临床重要差异。
