评估扁平苔藓中现有健康相关生活质量测量方法的适用性。
Evaluating the Appropriateness of Existing Health-Related Quality of Life Measures in Lichen Planus.
作者信息
Mahon-Smith Aoife, Skingley George, Ayala-Nunes Lara, Batish Anjali, Sharp Rosie, Naujoks Christel, Schruf Eva, Compagno Nicolò, Moreno Santiago G
机构信息
Patient-Centered Outcomes, Adelphi Values Ltd, Bollington, Cheshire, UK.
Novartis Pharma AG, Basel, Switzerland.
出版信息
Dermatol Ther (Heidelb). 2023 Nov;13(11):2817-2837. doi: 10.1007/s13555-023-00990-4. Epub 2023 Oct 5.
INTRODUCTION
Lichen planus (LP) is an inflammatory skin disorder that can present in various forms across the body, including lesions on the skin (cutaneous LP [CLP]), scalp (lichen planopilaris [LPP]) and mucosal regions (mucosal LP [MLP]). Several existing patient-reported outcome measures (PROMs) were identified for potential use in LP clinical development programs. This study aimed to assess the content validity and psychometric measurement properties of the Dermatology Life Quality Index (DLQI), Epworth Sleepiness Scale (ESS), Scalpdex and Oral Lichen Planus Symptom Severity Measure (OLPSSM) in an LP population.
METHODS
Patients completed the PROs at various time points as part of an international Phase 2 clinical study in adults with MLP (n = 37), LPP (n = 37) and CLP (n = 37). Test-retest reliability, construct validity and sensitivity to change were assessed. In addition, qualitative cognitive debriefing interviews were conducted with adults with MLP (n = 20), LPP (n = 19) and CLP (n = 19) in the USA and Germany to examine the PROM content validity.
RESULTS
The DLQI demonstrated adequate reliability and validity, although its ability to detect change was modest and most items were considered not relevant in qualitative interviews. The ESS had good reliability but limited evidence of validity and ability to detect change. Conceptual relevance varied according to the qualitative interview data. The Scalpdex was miscellaneous across domains, but the 'Symptoms' domain performed well overall. Overall, Scalpdex concepts were reported as relevant by most LPP patients interviewed. The OLPSSM demonstrated good psychometric properties and strong evidence of content validity.
CONCLUSIONS
The psychometric and qualitative findings support the use of the OLPSSM and Scalpdex within specific LP subtypes but cautioned use of the DLQI. Administration of the ESS is not recommended in LP because of its poor psychometric performance. Given these limitations, further validation of non-specific disease measures is needed and/or the development of additional LP-specific PROMs.
TRIAL REGISTRATION
NCT04300296.
引言
扁平苔藓（LP）是一种炎症性皮肤病，可在全身以多种形式出现，包括皮肤病变（皮肤扁平苔藓[CLP]）、头皮病变（扁平苔藓性毛发角化病[LPP]）和黏膜区域病变（黏膜扁平苔藓[MLP]）。已确定了几种现有的患者报告结局指标（PROMs）可用于LP临床开发项目。本研究旨在评估皮肤病生活质量指数（DLQI）、爱泼华嗜睡量表（ESS）、头皮指数（Scalpdex）和口腔扁平苔藓症状严重程度量表（OLPSSM）在LP患者群体中的内容效度和心理测量特性。
方法
作为一项针对成年MLP患者（n = 37）、LPP患者（n = 37）和CLP患者（n = 37）的国际2期临床研究的一部分，患者在不同时间点完成了这些PROMs。评估了重测信度、结构效度和对变化的敏感性。此外，在美国和德国对成年MLP患者（n = 20）、LPP患者（n = 19）和CLP患者（n = 19）进行了定性认知反馈访谈，以检查PROM的内容效度。
结果
DLQI显示出足够的信度和效度，尽管其检测变化的能力一般，且在定性访谈中大多数项目被认为不相关。ESS具有良好的信度，但效度和检测变化能力的证据有限。根据定性访谈数据，概念相关性各不相同。Scalpdex在各领域情况不一，但“症状”领域总体表现良好。总体而言，大多数接受访谈的LPP患者报告Scalpdex概念相关。OLPSSM显示出良好的心理测量特性和较强的内容效度证据。
结论
心理测量和定性研究结果支持在特定LP亚型中使用OLPSSM和Scalpdex，但对DLQI的使用需谨慎。由于其心理测量性能较差，不建议在LP中使用ESS。鉴于这些局限性，需要对非特异性疾病测量指标进行进一步验证和/或开发更多LP特异性PROMs。
试验注册
NCT04300296。
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