McKee Stephanie, Xenakis Jason, Makin Harriet, Marshall Chris, Winnette Randall, Aggarwal Rohit, Knight Sarah
Clinical Outcomes Assessment, Clarivate, London, UK.
Pfizer Inc, New York, USA.
Dermatol Ther (Heidelb). 2024 Oct;14(10):2771-2785. doi: 10.1007/s13555-024-01266-1. Epub 2024 Sep 12.
Dermatomyositis (DM) is a rare and severely debilitating autoimmune disease that can affect children and adults; however, there is little understanding of the patient-reported experience and uncertainty around validated clinical outcomes assessments (COAs) that could measure changes in the condition during clinical trials of new treatments.
The aim of this study was to understand the patient experience of DM, with a focus on its cutaneous manifestations, to describe the patient experience and determine the suitability of existing COA measures.
Adult (≥ 18 years) patients (N = 28) with severe active cutaneous manifestations of DM were interviewed. In the 90-min interviews, open-ended questions and probes were used to elicit descriptions of key clinical manifestations and patients' experiences of DM, including the symptoms and impacts on their daily lives and wellbeing.
Patients reported 13 different skin manifestations of DM. The most common were rash (n = 28, 100%), itch (n = 28, 100%), dry skin (n = 23, 82%), and swelling of the skin (n = 17, 61%). The head and face, followed by hands, were perceived as the most bothersome body areas affected by skin manifestations, because they are exposed and visible to themselves and other people. All patients (n = 28, 100%) reported at least one impact of DM, which varied greatly between patients, but included emotional, psychological, cognitive, and physical impacts, and those affecting daily life, such as work and sleep. Over half of the patients (n = 19, 67%) reported that their daily activities were impacted by DM.
The qualitative interviews with patients revealed that the presentation of DM manifestations is highly variable but affects patients' emotional wellbeing, physical activities, and daily life significantly.
皮肌炎（DM）是一种罕见且严重致残的自身免疫性疾病，可影响儿童和成人；然而，对于患者报告的体验以及在新治疗方法的临床试验中能够衡量病情变化的经过验证的临床结局评估（COA）方面，人们了解甚少。
本研究的目的是了解皮肌炎患者的体验，重点关注其皮肤表现，描述患者体验并确定现有COA测量方法的适用性。
对患有严重活动性皮肌炎皮肤表现的成年（≥18岁）患者（N = 28）进行了访谈。在90分钟的访谈中，使用开放式问题和追问来引出关键临床表现的描述以及患者对皮肌炎的体验，包括症状及其对日常生活和幸福感的影响。
患者报告了13种不同的皮肌炎皮肤表现。最常见的是皮疹（n = 28，100%）、瘙痒（n = 28，100%）、皮肤干燥（n = 23，82%）和皮肤肿胀（n = 17，61%）。头面部，其次是手部，被认为是受皮肤表现影响最困扰的身体部位，因为这些部位对患者自己和他人来说都是暴露且可见的。所有患者（n = 28，100%）都报告了皮肌炎至少一种影响，患者之间差异很大，但包括情感、心理、认知和身体方面的影响，以及那些影响日常生活的方面，如工作和睡眠。超过一半的患者（n = 19，67%）报告他们的日常活动受到皮肌炎的影响。
对患者的定性访谈表明，皮肌炎表现的呈现高度可变，但对患者的情绪健康、身体活动和日常生活有显著影响。
