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成人特应性皮炎和慢性手部湿疹的患者报告结局测量。

Patient-Reported Outcome Measures in Atopic Dermatitis and Chronic Hand Eczema in Adults.

机构信息

RTI Health Solutions, 3040 Cornwallis Drive, PO Box 12194, Research Triangle Park, NC, 27709, USA.

Leo Pharma, Ballerup, Denmark.

出版信息

Patient. 2019 Oct;12(5):445-459. doi: 10.1007/s40271-019-00373-y.

Abstract

Patient-reported outcome measures (PROMs) provide an important complement to physician-assessed clinical outcome measures in dermatologic diseases such as atopic dermatitis (AD) and chronic hand eczema (CHE). AD and CHE are chronic and relapsing inflammatory skin conditions that often co-occur. While both diseases result in various signs and symptoms that are burdensome and can negatively affect patients' lives, there may be distinct differences in the signs, symptoms, burden, and health-related quality of life (HRQOL) impact of these diseases. The objective of this study was to identify and evaluate PROMs used in studies of AD and CHE. The aim was to explore the assessment of key symptoms and impacts, and identify any gaps in the measures in use. A structured review of the PubMed database was conducted to identify PROMs used or developed for use in AD or CHE. The Dermatology Life Quality Index (DLQI), the Pruritus/Itch Numeric Rating Scale (NRS), the Patient-Oriented Eczema Measure (POEM), and the Quality of Life in Hand Eczema Questionnaire (QOLHEQ) were identified and reviewed in detail. With these measures, the AD and CHE symptoms and impacts most commonly evaluated in the literature include dermatology-related HRQOL in the domains of symptoms and feelings, daily activities, leisure, work and school, personal relationships, and adverse effects; pruritus; sleep disturbance; AD-specific symptoms (dryness, itching, flaking, cracking, bleeding, and weeping/oozing); and CHE-specific symptoms (pain, itch, fissuring, redness, bleeding, and dryness). A review of regulatory labels of drugs approved for AD by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) found that, among the four measures reviewed, the Pruritus NRS was included in the FDA and EMA labels for dupilumab, the DLQI was included in the EMA labels for dupilumab and tacrolimus, and the POEM was included in the EMA label for dupilumab. Key symptoms of AD (e.g. itching, flaking, cracking) and CHE (e.g. pain, itching, fissuring) are increasingly being assessed with PROMs; however, primary endpoints in clinical trials are often based on clinician-reported outcome measures. As therapeutic strategies in dermatology are targeted at specific dermatologic symptoms and diseases affecting specific sites (e.g. CHE), future research should explore patients' experiences with these symptoms and sites and the changes with treatment that are most meaningful to them.

摘要

患者报告结局测量(PROMs)为皮肤病学疾病(如特应性皮炎[AD]和慢性手部湿疹[CHE])的医生评估临床结局测量提供了重要的补充。AD 和 CHE 是慢性复发性炎症性皮肤疾病,常同时发生。虽然这两种疾病都会导致各种令人不适的症状和体征,且可能对患者的生活产生负面影响,但这些疾病的体征、症状、负担和健康相关生活质量(HRQOL)的影响可能存在明显差异。本研究的目的是确定并评估用于 AD 和 CHE 研究的 PROMs。目的是探讨关键症状和影响的评估,并确定使用中的测量方法是否存在差距。对 PubMed 数据库进行了系统回顾,以确定用于 AD 或 CHE 的 PROMs。皮肤病生活质量指数(DLQI)、瘙痒/瘙痒数字评定量表(NRS)、患者导向的湿疹测量(POEM)和手部湿疹生活质量问卷(QOLHEQ)被确定并进行了详细审查。这些措施中,AD 和 CHE 最常被文献评估的症状和影响包括皮肤科相关 HRQOL 的症状和感觉、日常活动、休闲、工作和学校、人际关系和不良反应;瘙痒;睡眠障碍;AD 特定症状(干燥、瘙痒、鳞屑、皲裂、出血和渗出/渗液);和 CHE 特定症状(疼痛、瘙痒、皲裂、发红、出血和干燥)。对美国食品和药物管理局(FDA)和欧洲药品管理局(EMA)批准用于 AD 的药物监管标签的审查发现,在审查的四项措施中,瘙痒 NRS 被包含在 FDA 和 EMA 批准的度普利尤单抗标签中,DLQI 被包含在 EMA 批准的度普利尤单抗和他克莫司标签中,POEM 被包含在 EMA 批准的度普利尤单抗标签中。AD(如瘙痒、鳞屑、皲裂)和 CHE(如疼痛、瘙痒、皲裂)的主要症状越来越多地通过 PROMs 进行评估;然而,临床试验的主要终点通常基于医生报告的结局测量。随着皮肤病学的治疗策略针对特定的皮肤科症状和影响特定部位的疾病(如 CHE),未来的研究应该探索患者对这些症状和部位的体验以及对他们最有意义的治疗变化。

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