Ho Calvin N, Bahadori Lila, Yale Marc, Bishop Naomi, Roberts Caroline, Meyers Oren, Bailey Julie, Korman Neil J
Late-Stage Development, Respiratory and Immunology, BioPharmaceuticals R&D, AstraZeneca, 1 Medimmune Way, Gaithersburg, MD, 20878, USA.
International Pemphigus and Pemphigoid Foundation, Roseville, CA, USA.
Dermatol Ther (Heidelb). 2025 May 14. doi: 10.1007/s13555-025-01424-z.
Bullous pemphigoid (BP) is a chronic autoimmune blistering disorder that imposes a significant health-related quality of life burden on the lives of patients; however, there are limited data on patient experiences.
We conducted qualitative open-ended interviews of patients from the USA, Spain, and France, to understand BP symptoms and impacts, and develop a conceptual model. Adults with a confirmed BP diagnosis were included. Patients with pemphigus or drug-induced BP were excluded.
Thirty participants were recruited. The mean (standard deviation) age was 63.6 (16.0) years. Eleven signs and symptoms and 24 impacts were reported; six signs and symptoms, and 13 impacts affected ≥ 50% of participants with an average disturbance rating of ≥ 5 out of 10 (scale of 0 to 10 [0, not disturbing; 10, extremely disturbing]). All participants reported typical signs and symptoms of classic and/or mucosal BP. Overall, five of six salient symptoms and four of 13 salient impacts had an average disturbance rating of ≥ 7.5/10, indicating that BP is highly burdensome. Regarding corticosteroids, participants expressed a strong desire to avoid taking them again, due to the associated unwanted side effects.
The conceptual model presented here can be the basis for endpoint selection in studies of new therapies for BP.
大疱性类天疱疮（BP）是一种慢性自身免疫性水疱性疾病，给患者的生活带来了与健康相关的显著生活质量负担；然而，关于患者经历的数据有限。
我们对来自美国、西班牙和法国的患者进行了定性开放式访谈，以了解BP的症状和影响，并建立一个概念模型。纳入确诊为BP的成年人。排除患有天疱疮或药物性BP的患者。
招募了30名参与者。平均（标准差）年龄为63.6（16.0）岁。报告了11种体征和症状以及24种影响；6种体征和症状以及13种影响影响了≥50%的参与者，平均干扰评分为≥10分制中的5分（0至10分制[0，无干扰；10，极度干扰]）。所有参与者均报告了经典型和/或黏膜型BP的典型体征和症状。总体而言，六种显著症状中的五种以及13种显著影响中的四种平均干扰评分为≥7.5/10，表明BP负担很重。关于皮质类固醇，由于相关的不良副作用，参与者表达了强烈的不再服用它们的愿望。
这里提出的概念模型可作为BP新疗法研究中终点选择的基础。
