Patient-reported outcomes and quality of life in recessive dystrophic epidermolysis bullosa: A global cross-sectional survey
Patient-reported outcomes and quality of life in recessive dystrophic epidermolysis bullosa: A global cross-sectional survey
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DOI：
10.1016/j.jaad.2020.03.028
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发表时间：
2021-10-13
影响因子：
13.8
通讯作者：
Tang, Jean Y.
中科院分区：
文献类型：
--
作者：
Eng, Victor A.;Solis, Daniel C.;Tang, Jean Y.
背景：隐性营养不良性大疱性表皮病（RDEB）患者存在一系列皮肤病严重程度。目的：描述RDEB患者的患者报告结局和生活质量（QOL）。.方法：通过全球EBCare登记研究对RDEB患者进行横断面研究。患者报告的结局包括皮肤病严重程度、伤口特征、疼痛、瘙痒、皮外症状和药物治疗。采用经验证的大疱性表皮病患者生活质量量表测量生活质量。结果：共有85例RDEB患者报告了1226处伤口（937处复发性伤口和289处慢性开放性伤口）。总体皮肤病严重程度自我报告为轻度（26%; 22/83）、中度（48%; 40/ 83）或重度（25%; 21/83）。皮肤病严重程度的恶化与较大的伤口、阿片类药物使用增加、贫血、胃造口管使用、感染、骨质疏松症和鳞状细胞癌显著相关。伤口尺寸越大，生活质量评分越差。局限性：所有数据均来自在线大疱性表皮松解症患者登记处的自我报告。结论：这项研究表明，在RDEB参与者中，较大的伤口尺寸与皮肤病严重程度恶化和生活质量之间存在显著相关性。皮肤病严重程度恶化与关键临床表现显著相关。这些结果表明，RDEB患者能够自我报告他们的皮肤病严重程度和伤口。（《美国皮肤科学院杂志》2021;85：1161-7。）
Background: A spectrum of skin disease severity exists in patients with recessive dystrophic epidermolysis bullosa (RDEB). Objective: To characterize the patient-reported outcomes and quality of life (QOL) in patients with RDEB. . Methods: A cross-sectional study of patients with RDEB surveyed through the global EBCare Registry. Patient-reported outcomes included skin disease severity, wound characteristics, pain, itch, extracutaneous symptoms, and medications. QOL was measured by using the validated Quality of Life in Epidermolysis Bullosa instrument. Results: A total of 85 patients with RDEB reported 1226 wounds (937 recurrent wounds and 289 chronic open wounds). Overall skin disease severity was self-reported as mild (26%; 22/83), moderate (48%; 40/ 83), or severe (25%; 21/83). Worsening skin disease severity was significantly associated with larger wounds, increased opiate use, anemia, gastrostomy tube use, infections, osteoporosis, and squamous cell carcinoma. Larger wound size was associated with worse quality of life scores. Limitations: All data were self-reported from an online epidermolysis bullosa patient registry. Conclusions: This study shows a significant correlation between larger wound size with worsening skin disease severity and quality of life in participants with RDEB. Worsening skin disease severity significantly correlated with key clinical manifestations. These results show that patients with RDEB are able to self report their skin disease severity and wounds. ( J Am Acad Dermatol 2021;85:1161-7.)