Abstract：
BACKGROUND: Dermatomyositis (DM) is a rare systemic autoimmune disease characterized by a distinctive debilitating skin rash and skeletal muscle weakness. It is unclear if existing clinical outcome assessment (COA) measures include the concepts of priority to patients and those necessary to fully capture improvements in the active cutaneous manifestations of DM. This study aimed to develop the Cutaneous Dermatomyositis Investigator Global Assessment (CDM-IGA), a de novo IGA, for use in clinical trials of adult DM.
METHODS: Eight DM clinical experts participated in 60-min qualitative interviews consisting of concept elicitation and cognitive debriefing methodologies. Concept elicitation comprised open-ended questions with follow-up probes to explore clinicians\' experiences of treating patients with DM, the impact of symptoms on patients\' quality of life, and the severity levels of disease characteristics to explore DM progression. Cognitive debriefing required the clinical experts to perform a review of the CDM-IGA, designed to assess the severity of cutaneous disease activity of DM. After the interviews, a consensus meeting with three clinical experts was held to agree on any outstanding issues relating to the CDM-IGA.
RESULTS: The CDM-IGA was iteratively developed using the opinions of nine clinical experts. Feedback provided by all clinicians agreed that erythema was the main active cutaneous manifestation of DM and should be the primary characteristic on the CDM-IGA, split by erythema color and extent. To determine cutaneous disease severity, experts suggested adding a metric called secondary changes, which combined erosion/ulceration and lichenification, which could modify the patient\'s final score. Three clinical experts suggested that a photo-guide to support assessments of erythema across different skin tones could be beneficial.
CONCLUSIONS: A novel CDM-IGA was developed for use with adult patients with DM in clinical trials, based on an iterative development process that combined qualitative feedback from clinical experts of DM and importantly adult patients living with DM.
METHODS: Eight DM clinical experts participated in 60-min qualitative interviews consisting of concept elicitation and cognitive debriefing methodologies. Concept elicitation comprised open-ended questions with follow-up probes to explore clinicians\' experiences of treating patients with DM, the impact of symptoms on patients\' quality of life, and the severity levels of disease characteristics to explore DM progression. Cognitive debriefing required the clinical experts to perform a review of the CDM-IGA, designed to assess the severity of cutaneous disease activity of DM. After the interviews, a consensus meeting with three clinical experts was held to agree on any outstanding issues relating to the CDM-IGA.
RESULTS: The CDM-IGA was iteratively developed using the opinions of nine clinical experts. Feedback provided by all clinicians agreed that erythema was the main active cutaneous manifestation of DM and should be the primary characteristic on the CDM-IGA, split by erythema color and extent. To determine cutaneous disease severity, experts suggested adding a metric called secondary changes, which combined erosion/ulceration and lichenification, which could modify the patient\'s final score. Three clinical experts suggested that a photo-guide to support assessments of erythema across different skin tones could be beneficial.
CONCLUSIONS: A novel CDM-IGA was developed for use with adult patients with DM in clinical trials, based on an iterative development process that combined qualitative feedback from clinical experts of DM and importantly adult patients living with DM.
摘要：
背景：皮肌炎（DM）是一种罕见的全身性自身免疫性疾病，其特征是独特的衰弱性皮疹和骨骼肌无力。目前尚不清楚现有的临床结果评估（COA）措施是否包括优先考虑患者的概念以及完全捕获DM活动性皮肤表现改善所必需的概念。本研究旨在开发皮肤皮肌炎研究者全球评估（CDM-IGA），一个从头的IGA，用于成人DM的临床试验。
方法：八名DM临床专家参加了60分钟的定性访谈，包括概念启发和认知汇报方法。概念启发包括开放式问题和随访探针，以探索临床医生治疗DM患者的经验，症状对患者生活质量的影响，和疾病的严重程度特征来探讨DM的进展。认知汇报要求临床专家对CDM-IGA进行审查，旨在评估DM皮肤疾病活动的严重程度。面试后,举行了与三名临床专家的协商一致会议，就与CDM-IGA有关的任何未决问题达成一致。
结果：使用九位临床专家的意见迭代开发了CDM-IGA。所有临床医生提供的反馈都认为红斑是DM的主要活性皮肤表现，应该是CDM-IGA的主要特征，按红斑的颜色和程度分开。为了确定皮肤疾病的严重程度，专家建议增加一个叫做二次变化的指标，结合了侵蚀/溃疡和苔藓化，这可以修改病人的最终分数。三位临床专家建议，支持不同肤色红斑评估的照片指南可能是有益的。
结论：开发了一种新型CDM-IGA，用于成年DM患者的临床试验，基于迭代开发过程，该过程结合了DM临床专家和重要的成年DM患者的定性反馈。
方法：八名DM临床专家参加了60分钟的定性访谈，包括概念启发和认知汇报方法。概念启发包括开放式问题和随访探针，以探索临床医生治疗DM患者的经验，症状对患者生活质量的影响，和疾病的严重程度特征来探讨DM的进展。认知汇报要求临床专家对CDM-IGA进行审查，旨在评估DM皮肤疾病活动的严重程度。面试后,举行了与三名临床专家的协商一致会议，就与CDM-IGA有关的任何未决问题达成一致。
结果：使用九位临床专家的意见迭代开发了CDM-IGA。所有临床医生提供的反馈都认为红斑是DM的主要活性皮肤表现，应该是CDM-IGA的主要特征，按红斑的颜色和程度分开。为了确定皮肤疾病的严重程度，专家建议增加一个叫做二次变化的指标，结合了侵蚀/溃疡和苔藓化，这可以修改病人的最终分数。三位临床专家建议，支持不同肤色红斑评估的照片指南可能是有益的。
结论：开发了一种新型CDM-IGA，用于成年DM患者的临床试验，基于迭代开发过程，该过程结合了DM临床专家和重要的成年DM患者的定性反馈。
