Psychometric Evaluation of the Facial and Total Vitiligo Area Scoring Index Instruments in the TRuE-V Phase 3 Studies.

IF 3.5 3区医学 Q1 DERMATOLOGY

Dermatology and Therapy Pub Date : 2024-08-01 Epub Date: 2024-07-30 DOI:10.1007/s13555-024-01223-y

Kristen Bibeau, Kathleen Butler, Mingyue Wang, Konstantina Skaltsa, Iltefat H Hamzavi

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引用次数: 0

Abstract

Introduction: This study reports psychometric testing of the facial and total Vitiligo Area Scoring Index quantitative clinical instruments (F-VASI [range: 0-3], T-VASI [range: 0-100], respectively) using data from two phase 3 randomized, vehicle-controlled studies of ruxolitinib cream (TRuE-V1/TRuE-V2), the largest vitiligo trials conducted to date. Because VASI assessment is required by regulatory authorities, we evaluated the psychometric properties of the VASI instruments and confirmed thresholds for clinically meaningful change.

Methods: The TRuE-V1/TRuE-V2 full analysis set population included 652 patients (≥ 12 years old with nonsegmental vitiligo affecting ≤ 10% total body surface area, F-VASI ≥ 0.5, and T-VASI ≥ 3 at baseline). Data collected using the facial and total Patient Global Impression of Change-Vitiligo (PaGIC-V) and Physician's Global Vitiligo Assessment (PhGVA) scales were used as anchors to assess F-VASI and T-VASI for reliability, validity, sensitivity to change, and clinically meaningful change.

Results: Median F-VASI and T-VASI scores were 0.70 and 6.76, respectively, at baseline, decreasing to 0.48 and 4.80 at week 24. Test-retest reliability was excellent between screening and baseline for F-VASI (intraclass correlation coefficient [ICC]: 0.943) and T-VASI (ICC: 0.945). Among stable patients per PaGIC-V and PhGVA, reliability was moderate to good for both F-VASI (ICC: 0.891 and 0.739, respectively) and T-VASI (ICC: 0.768 and 0.686). F-VASI and T-VASI differentiated well among PhGVA categories mild/moderate/severe at baseline and week 24. Both VASI instruments detected changes assessed by correlations with PaGIC-V scores at week 24 (F-VASI, r = 0.610; T-VASI, r = 0.512) and changes in PhGVA scores from baseline to week 24 (F-VASI, r = 0.501; T-VASI, r = 0.344). Thresholds for clinically meaningful improvement per PaGIC-V and PhGVA were 0.38-0.60 for F-VASI and 1.69-3.88 for T-VASI.

Conclusions: Data from the TRuE-V1/TRuE-V2 studies confirmed that F-VASI and T-VASI are reliable, valid, and responsive to change, with defined clinically meaningful change from baseline in patients with nonsegmental vitiligo.

Trial registration: The original studies were registered at ClinicalTrials.gov: NCT04052425/NCT04057573.

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TRuE-V 3 期研究中面部和全身白癜风面积评分指数工具的心理计量学评估。

导言：本研究报告了对面部和总白癜风面积评分指数定量临床工具（F-VASI [范围：0-3]，T-VASI [范围：0-100]，分别为[0-3]和[0-100]）的心理测试，测试使用的数据来自两项鲁索利替尼乳膏（TRuE-V1/TRuE-V2）的3期随机、载体对照研究，这是迄今为止进行的最大规模的白癜风试验。由于监管机构要求进行VASI评估，因此我们评估了VASI工具的心理测量特性，并确认了有临床意义变化的阈值：TRuE-V1/TRuE-V2完整分析组人群包括652名患者（年龄≥12岁，非节段性白癜风累及体表总面积≤10%，基线时F-VASI≥0.5，T-VASI≥3）。以面部和全身白癜风患者总体变化印象量表（PaGIC-V）和医生总体白癜风评估量表（PhGVA）收集的数据为锚，评估F-VASI和T-VASI的可靠性、有效性、对变化的敏感性和有临床意义的变化：基线时 F-VASI 和 T-VASI 的中位数分别为 0.70 分和 6.76 分，第 24 周时分别降至 0.48 分和 4.80 分。F-VASI（类内相关系数 [ICC]：0.943）和T-VASI（ICC：0.945）在筛查和基线之间的重复测试可靠性极佳。在 PaGIC-V 和 PhGVA 的稳定期患者中，F-VASI（ICC 分别为 0.891 和 0.739）和 T-VASI （ICC 分别为 0.768 和 0.686）的可靠性为中等至良好。在基线和第 24 周时，F-VASI 和 T-VASI 能很好地区分 PhGVA 的轻度/中度/重度类别。通过与第 24 周时 PaGIC-V 评分的相关性（F-VASI，r = 0.610；T-VASI，r = 0.512）以及 PhGVA 评分从基线到第 24 周的变化（F-VASI，r = 0.501；T-VASI，r = 0.344），两种 VASI 工具都能检测到变化。F-VASI的PaGIC-V和PhGVA有临床意义的改善阈值为0.38-0.60，T-VASI为1.69-3.88：TRuE-V1/TRuE-V2研究的数据证实，F-VASI和T-VASI是可靠、有效的，对变化反应灵敏，在非节段性白癜风患者中，与基线相比有明确的临床意义的变化：原始研究已在 ClinicalTrials.gov 注册：NCT04052425/NCT04057573.

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来源期刊

Dermatology and Therapy Medicine-Dermatology

CiteScore

6.00

自引率

8.80%

发文量

187

审稿时长

6 weeks

期刊介绍： Dermatology and Therapy is an international, open access, peer-reviewed, rapid publication journal (peer review in 2 weeks, published 3–4 weeks from acceptance). The journal is dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of dermatological therapies. Studies relating to diagnosis, pharmacoeconomics, public health and epidemiology, quality of life, and patient care, management, and education are also encouraged. Areas of focus include, but are not limited to all clinical aspects of dermatology, such as skin pharmacology; skin development and aging; prevention, diagnosis, and management of skin disorders and melanomas; research into dermal structures and pathology; and all areas of aesthetic dermatology, including skin maintenance, dermatological surgery, and lasers. The journal is of interest to a broad audience of pharmaceutical and healthcare professionals and publishes original research, reviews, case reports/case series, trial protocols, and short communications. Dermatology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an International and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of quality research, which may be considered of insufficient interest by other journals. The journal appeals to a global audience and receives submissions from all over the world.