McKee Stephanie, Xenakis Jason, Makin Harriet, Marshall Chris, Winnette Randall, Aggarwal Rohit, Knight Sarah L
Clinical Outcomes Assessment, Clarivate, London, UK.
Pfizer Inc, New York, USA.
Dermatol Ther (Heidelb). 2024 Aug;14(8):2127-2138. doi: 10.1007/s13555-024-01220-1. Epub 2024 Jul 8.
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.
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.
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.
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.
皮肌炎（DM）是一种罕见的系统性自身免疫性疾病，其特征为独特的使人衰弱的皮疹和骨骼肌无力。目前尚不清楚现有的临床结局评估（COA）措施是否涵盖了对患者而言的优先事项概念以及全面捕捉DM活动性皮肤表现改善情况所需的概念。本研究旨在开发一种全新的皮肌炎研究者整体评估（CDM-IGA），用于成人DM的临床试验。
八位DM临床专家参与了为期60分钟的定性访谈，访谈采用概念引出和认知反馈方法。概念引出包括开放式问题及后续追问，以探究临床医生治疗DM患者的经验、症状对患者生活质量的影响以及疾病特征的严重程度水平，从而探索DM的进展情况。认知反馈要求临床专家对旨在评估DM皮肤疾病活动严重程度的CDM-IGA进行审查。访谈结束后，与三位临床专家举行了共识会议，就与CDM-IGA相关的任何未决问题达成一致。
CDM-IGA是在九位临床专家的意见基础上经过反复开发而成。所有临床医生提供的反馈一致认为，红斑是DM主要的活动性皮肤表现，应作为CDM-IGA的主要特征，并按红斑颜色和范围进行划分。为确定皮肤疾病的严重程度，专家们建议增加一个名为继发性改变的指标，该指标将糜烂/溃疡和苔藓化合并在一起，可修正患者的最终得分。三位临床专家建议，提供一份用于支持评估不同肤色红斑情况的照片指南可能会有所帮助。
基于一个反复开发过程，结合了DM临床专家尤其是成年DM患者的定性反馈，开发出了一种新型的CDM-IGA，用于成人DM患者的临床试验。
