背景:广泛性重症肌无力(gMG)是一种罕见的自身免疫性疾病。gMG的症状多种多样,对患者影响的理解是有限的。这项定性研究旨在深入探索gMG患者的日常体验。
方法:回顾了已发表的定性研究,以确定最重要的体征,症状,以及与gMG患者体验相关的功能影响。对临床医生和成年gMG患者进行了半结构化混合概念启发访谈(允许自发产生疾病概念)和认知汇报访谈(评估现有疾病评估的有效性)。迹象,症状,并对影响进行了审查,以了解哪些是最显著的(即,至少50%的患者报告,干扰等级为5或更高[10分数字量表]);还评估了概念饱和度。更新了疾病概念模型。现有的临床结果评估(COA),捕捉患者的感受,函数,和生存进行了评估。
结果:与文献相比,对患者(n=24)的访谈发现了7种新的体征和症状以及37种新的影响。达到概念饱和。患者确定为最重要(显着)的体征和症状是呼吸急促,一般疲劳,手臂肌肉无力,腿,脖子,发音困难,构音障碍,吞咽困难的液体,窒息,和热敏感性。患者识别的突出影响是工作生活,抑郁症,行走困难,梳理头发,淋浴,刷牙,吃,个人关系,家庭生活,参与社会活动。临床医生认为眼,呼吸,吞咽,演讲/谈话,和肢体功能是gMG的主要临床表现。患者和临床医生发现临床结果评估(COA)在概念上是相关和全面的。
结论:这项研究提供了对gMG体征的整体理解,症状,以及患者所经历的影响,正如患者和临床医生所观察到的。gMG的概念模型突出了标志的范围,症状,并影响成年gMG患者在日常生活中的经历,强调人文影响和未满足的需求。
BACKGROUND: Generalized myasthenia gravis (gMG) is a rare autoimmune disease. Symptoms of gMG are diverse, and understanding of their impact on patients is limited. This qualitative study aimed to provide an in-depth exploration of patients\' daily experiences of gMG.
METHODS: Published qualitative studies were reviewed to identify the most important signs, symptoms, and functional impacts related to the patient experience in gMG. Semi-structured hybrid concept elicitation interviews (allowing spontaneous generation of disease concepts) and cognitive debriefing interviews (assessing the validity of existing disease assessments) were conducted with clinicians and adult patients with gMG. Signs, symptoms, and impacts were reviewed to understand which were most salient (i.e., reported by at least 50% of patients, with disturbance rating 5 or higher [10-point numeric scale]); concept saturation was also assessed. The disease conceptual model was updated. Existing clinical outcomes assessments (COAs) that capture how patients feel, function, and survive were assessed.
RESULTS: Interviews with patients (n = 24) identified seven new signs and symptoms and 37 new impacts compared with the literature. Concept saturation was reached. Signs and symptoms identified by patients as most important (salient) were shortness of breath, general fatigue, muscle weakness of arms, legs, and neck, dysphonia, dysarthria, trouble swallowing liquids, choking, and heat sensitivity. Patient-identified salient impacts were work life, depression, difficulty walking, grooming hair, showering, and brushing teeth, eating, personal relationships, family life, and participating in social activities. Clinicians considered ocular, respiratory, swallowing, speech/talking, and extremity function as key clinical manifestations of gMG. Patients and clinicians found clinical outcome assessments (COAs) to be conceptually relevant and comprehensive.
CONCLUSIONS: This research provides a holistic understanding of gMG signs, symptoms, and impacts experienced by patients, as observed by patients and clinicians. The conceptual model of gMG highlights the range of signs, symptoms, and impacts that adult patients with gMG experience in their everyday lives, emphasizing the humanistic impact and unmet needs.