Andrew Lloyd, Daniel Aggio, Caleb Dixon, Ernest H Law, Thomas Price
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Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss.</p><p><strong>Objectives: </strong>This study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C).</p><p><strong>Methods: </strong>Data from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed.</p><p><strong>Results: </strong>Data were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3-61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO.</p><p><strong>Conclusions: </strong>The EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. 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引用次数: 0
摘要
背景：对于许多健康技术评估的决策者来说，EQ-5D-5L 是衡量健康相关生活质量 (HRQL) 的标准指标。然而，有证据表明该指标在某些疾病领域存在局限性，包括皮肤科。斑秃（AA）对患者的 HRQL 影响很大，部分原因是脱发对患者的情绪造成了影响：本研究探讨了 EQ-5D-5L 在 AA 患者中的心理计量特性，并参考了短表 36 版 2（SF-36v2）、脱发患者优先结果（AAPPO）、脱发严重程度工具（SALT）和患者全球变化印象（PGI-C）：分析了参加利特西替尼 ALLEGRO-2b/3 试验（NCT03732807）的 AA 患者的数据。参与者在48周的随访期间完成了AAPPO测量（一种评估情绪症状和活动限制的AA专用测量）、PGI-C、EQ-5D-5L和SF-36v2。头皮脱发程度使用 SALT 进行评估。对上限效应、已知群体有效性、收敛有效性和响应性进行了检验。已知组别由 SALT 评分和 PGI-C 基线反应定义。此外，还进行了探索性因子分析：结果：612 名成年参与者提供了数据。在 EQ-5D-5L 中观察到了天花板效应（55.3%-61.2%），分析表明 EQ-5D 没有像 SF-36v2 那样捕捉到患者之间的重要差异。EQ-5D-5L 与 SALT 评分的相关性很弱，而 AAPPO 与脱发程度的相关性更强。与 EQ-5D-5L 相比，AAPPO 更能区分 SALT 和 PGI-C 所定义的已知组别。探索性因素分析表明，与 AAPPO 相比，EQ-5D-5L 在内容有效性方面存在局限性：结论：EQ-5D-5L 可能无法充分衡量 AA 对患者 HRQL 造成的负担。对 AA 负担的不敏感性表明，EQ-5D-5L 可能无法衡量头发再生带来的治疗相关益处。如果其他测量方法的数据被证明更具相关性，则可以考虑使用这些数据。
Psychometric Properties of the EQ-5D-5L in Patients with Alopecia Areata.
Background: For many decision makers in Health Technology Assessment the EQ-5D-5L is the standard measure of health-related quality of life (HRQL). However, evidence has shown the limitations of the measure in certain disease areas, including dermatology. Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss.
Objectives: This study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C).
Methods: Data from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed.
Results: Data were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3-61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO.
Conclusions: The EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. Data from other measures could be considered if they are shown to be more relevant.
期刊介绍：
PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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