Abstract：
BACKGROUND: Generalized pustular psoriasis (GPP) is a rare, chronic, inflammatory skin disease associated with considerable patient burden. The Psoriasis Symptom Scale (PSS), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and pain-Visual Analogue Scale (pain-VAS) are patient-reported outcomes (PROs) that have not yet been validated in patients with GPP.
OBJECTIVE: To evaluate the psychometric properties of the PSS, FACIT-Fatigue and pain-VAS using data from Effisayil 1, a randomised trial of spesolimab in patients with moderate-to-severe GPP.
METHODS: Inter-item correlations and confirmatory factor analysis (CFA) were performed using Week 1 data. Internal consistency was assessed with Cronbach\'s α coefficient using baseline and Week 1 data. Test-retest reliability was assessed using intraclass correlation coefficients (ICCs); change data for the GPP Physician Global Assessment total score and pustulation subscore were used to define a stable population. Convergent validity was assessed at baseline and Week 1 using Spearman\'s rank-order correlations. Known-groups validity was measured by analysis of variance using Week 1 data. Ability to detect change from baseline to Week 1 was evaluated by analysis of covariance.
RESULTS: Inter-item and item-to-total correlations were moderate or strong for most PSS and FACIT-Fatigue items. CFA demonstrated the unidimensionality of the PSS and FACIT-Fatigue, with high factor loadings for most items (PSS range, 0.75-0.94; FACIT-Fatigue range, 0.11-0.93) and acceptable fit statistics. Both scores demonstrated internal consistency (Cronbach\'s α, 0.71 and 0.95, respectively). The PSS, FACIT-Fatigue and pain-VAS demonstrated test-retest reliability (ICCs ≥0.70) and good evidence of convergent validity. Furthermore, the PROs could differentiate between known groups of varying symptom severity (range, p < 0.0001-0.0225) and detect changes in symptom severity from baseline to Week 1 (range, p < 0.0001-0.0002).
CONCLUSIONS: Overall, these results support the reliability, validity and ability to detect change of the PSS, FACIT-Fatigue and pain-VAS as PROs in patients with GPP.
OBJECTIVE: To evaluate the psychometric properties of the PSS, FACIT-Fatigue and pain-VAS using data from Effisayil 1, a randomised trial of spesolimab in patients with moderate-to-severe GPP.
METHODS: Inter-item correlations and confirmatory factor analysis (CFA) were performed using Week 1 data. Internal consistency was assessed with Cronbach\'s α coefficient using baseline and Week 1 data. Test-retest reliability was assessed using intraclass correlation coefficients (ICCs); change data for the GPP Physician Global Assessment total score and pustulation subscore were used to define a stable population. Convergent validity was assessed at baseline and Week 1 using Spearman\'s rank-order correlations. Known-groups validity was measured by analysis of variance using Week 1 data. Ability to detect change from baseline to Week 1 was evaluated by analysis of covariance.
RESULTS: Inter-item and item-to-total correlations were moderate or strong for most PSS and FACIT-Fatigue items. CFA demonstrated the unidimensionality of the PSS and FACIT-Fatigue, with high factor loadings for most items (PSS range, 0.75-0.94; FACIT-Fatigue range, 0.11-0.93) and acceptable fit statistics. Both scores demonstrated internal consistency (Cronbach\'s α, 0.71 and 0.95, respectively). The PSS, FACIT-Fatigue and pain-VAS demonstrated test-retest reliability (ICCs ≥0.70) and good evidence of convergent validity. Furthermore, the PROs could differentiate between known groups of varying symptom severity (range, p < 0.0001-0.0225) and detect changes in symptom severity from baseline to Week 1 (range, p < 0.0001-0.0002).
CONCLUSIONS: Overall, these results support the reliability, validity and ability to detect change of the PSS, FACIT-Fatigue and pain-VAS as PROs in patients with GPP.
摘要：
背景：泛发性脓疱型银屑病（3GPP）是一种罕见的，慢性，炎症性皮肤病与相当大的患者负担相关。银屑病症状量表（PSS），慢性疾病治疗-疲劳的功能评估（FACIT-疲劳）和疼痛-视觉模拟量表（疼痛-VAS）是患者报告的结果（PRO），尚未在GMP患者中得到验证。
目的：为了评估PSS的心理测量学特性，FACIT-疲劳和疼痛-VAS使用来自Effisayil1的数据，该数据是spesolimab在中度至重度GMP患者中的随机试验。
方法：使用第1周数据进行项目间相关性和验证性因素分析（CFA）。使用基线和第1周数据用Cronbach'sα系数评估内部一致性。使用组内相关系数（ICC）评估了重测可靠性；使用了GMP医师全球评估总分和脓疱子得分的变化数据来定义稳定的人群。使用Spearman的等级顺序相关性在基线和第1周评估收敛有效性。通过使用第1周数据的方差分析来测量已知组的有效性。通过协方差分析评估检测从基线到第1周的变化的能力。
结果：对于大多数PSS和FACIT-疲劳项目，项目间和项目与总的相关性是中等或强的。CFA证明了PSS和FACIT-疲劳的一维性，对于大多数项目具有高因子载荷(PSS范围，0.75-0.94;FACIT-疲劳范围,0.11-0.93)和可接受的拟合统计数据。两个分数都表现出内部一致性(Cronbach'sα，分别为0.71和0.95）。PSS,FACIT-疲劳和疼痛-VAS表现出重测可靠性（ICC≥0.70）和收敛有效性的良好证据。此外，PRO可以区分不同症状严重程度的已知组(范围，p<0.0001-0.0225），并检测症状严重程度从基线到第1周的变化（范围，p<0.0001-0.0002)。
结论：总体而言，这些结果支持可靠性，检测PSS变化的有效性和能力，FACIT-疲劳和疼痛-VAS作为GMP患者的PRO。
目的：为了评估PSS的心理测量学特性，FACIT-疲劳和疼痛-VAS使用来自Effisayil1的数据，该数据是spesolimab在中度至重度GMP患者中的随机试验。
方法：使用第1周数据进行项目间相关性和验证性因素分析（CFA）。使用基线和第1周数据用Cronbach'sα系数评估内部一致性。使用组内相关系数（ICC）评估了重测可靠性；使用了GMP医师全球评估总分和脓疱子得分的变化数据来定义稳定的人群。使用Spearman的等级顺序相关性在基线和第1周评估收敛有效性。通过使用第1周数据的方差分析来测量已知组的有效性。通过协方差分析评估检测从基线到第1周的变化的能力。
结果：对于大多数PSS和FACIT-疲劳项目，项目间和项目与总的相关性是中等或强的。CFA证明了PSS和FACIT-疲劳的一维性，对于大多数项目具有高因子载荷(PSS范围，0.75-0.94;FACIT-疲劳范围,0.11-0.93)和可接受的拟合统计数据。两个分数都表现出内部一致性(Cronbach'sα，分别为0.71和0.95）。PSS,FACIT-疲劳和疼痛-VAS表现出重测可靠性（ICC≥0.70）和收敛有效性的良好证据。此外，PRO可以区分不同症状严重程度的已知组(范围，p<0.0001-0.0225），并检测症状严重程度从基线到第1周的变化（范围，p<0.0001-0.0002)。
结论：总体而言，这些结果支持可靠性，检测PSS变化的有效性和能力，FACIT-疲劳和疼痛-VAS作为GMP患者的PRO。
