Pandya Rachita, Dan Joshua, Kleitsch Julianne, Lim Darosa, White Barbara, Werth Victoria P
Corporal Micheal J. Crescenz Veterans Affairs Medical Center, Philadelphia, Pennsylvania, USA.
Department of Dermatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
JID Innov. 2024 Dec 9;5(2):100337. doi: 10.1016/j.xjidi.2024.100337. eCollection 2025 Mar.
The Total Improvement Score (TIS), which is used as the primary efficacy measure in dermatomyositis (DM) clinical trials, lacks a skin-specific measure. However, skin is a defining feature of DM. In this study, data were analyzed from the phase 3 trial of lenabasum in DM. Cutaneous Dermatomyositis Disease Area and Severity Index-Activity scores and all components of the TIS were collected at baseline and weeks 16, 28, 40, and 52. From these assessments, a composite outcome was developed, named Dermatomyositis Outcomes for Muscle and Skin, which includes certain components of the TIS and the Cutaneous Dermatomyositis Disease Area and Severity Index-Activity scores. The relative sensitivities of the TIS and Dermatomyositis Outcomes for Muscle and Skin to detect improvement in DM skin and muscle disease activity were analyzed. A total of 174 patients with DM were included, 82% were female, and 75% were White. Mean (SD) age was 51.9 (12.20) years. Treatment effect using the TIS ranged between 17.6 and 21.7 points for muscle and skin responders versus nonresponders across time points. The Dermatomyositis Outcomes for Muscle and Skin score displayed a statistically significantly greater treatment effect of 25.9-40.0 points for responders than for nonresponders, depending on the response assessed and the time point. Dermatomyositis Outcomes for Muscle and Skin is a more sensitive composite measure that reflects improvement from baseline in both skin and muscle disease activity, suggesting usefulness for use in future DM clinical trials.
总改善评分（TIS）在皮肌炎（DM）临床试验中用作主要疗效指标，但缺乏针对皮肤的特异性指标。然而，皮肤是DM的一个关键特征。在本研究中，分析了lenabasum治疗DM的3期试验数据。在基线以及第16、28、40和52周时收集皮肤性皮肌炎疾病面积和严重程度指数-活动度评分以及TIS的所有组成部分。基于这些评估，制定了一个综合结局指标，称为肌肉和皮肤皮肌炎结局指标，其中包括TIS的某些组成部分以及皮肤性皮肌炎疾病面积和严重程度指数-活动度评分。分析了TIS和肌肉和皮肤皮肌炎结局指标检测DM皮肤和肌肉疾病活动改善情况的相对敏感性。总共纳入了174例DM患者，其中82%为女性，75%为白人。平均（标准差）年龄为51.9（12.20）岁。在各个时间点，肌肉和皮肤有反应者与无反应者相比，使用TIS得出的治疗效果在17.6至21.7分之间。根据评估的反应和时间点，肌肉和皮肤皮肌炎结局指标评分显示，有反应者的治疗效果比无反应者在统计学上显著更高，为25.9 - 40.0分。肌肉和皮肤皮肌炎结局指标是一个更敏感的综合指标，反映了皮肤和肌肉疾病活动度相对于基线的改善情况，表明其在未来DM临床试验中具有应用价值。
