良性食管疾病的患者报告结局指标

Patient-Reported Outcome Measures in Benign Esophageal Disorders.

作者信息

Pandolfino John E, Fass Ronnie, Chan Walter W, Gyawali C Prakash

机构信息

Division of Gastroenterology and Hepatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA.

MetroHealth Medical Center and Case Western Reserve University, Cleveland, Ohio, USA.

出版信息

Am J Gastroenterol. 2025 Apr 7. doi: 10.14309/ajg.0000000000003467.

Abstract

Patient-reported outcome (PRO) measures are essential tools for assessing a patient's subjective experience related to disease and health. PROs measure symptom severity and evaluate treatment efficacy across a range of conditions at a particular point in time. Although PROs focusing on esophageal symptoms and esophageal hypervigilance exist, disease-specific PROs for commonly encountered benign esophageal disorders such as gastroesophageal reflux disease (GERD), eosinophilic esophagitis (EoE), and achalasia are limited. Most GERD-specific PROs fail to address the complete spectrum of GERD presentations and those that provide daily assessment are more suited for research. Similarly, many EoE-specific PROs were designed for clinical trials. Comprehensive instruments incorporating EoE symptoms, as well as endoscopic and histologic features of active inflammation and fibrostenotic changes are needed. The psychometric properties of the Eckardt Score used for achalasia have significant limitations, stemming primarily from the dominance of dysphagia in scoring. Newer achalasia-specific PROs attempt to overcome this by capturing nuanced patient experiences. Broader symptom PROs are often used to assess esophageal symptoms across the spectrum of benign esophageal disorders, including a PRO that assesses esophageal hypervigilance and symptom-specific anxiety. Future efforts should focus on creating user-friendly PROs that comprehensively evaluate not just clinical presentation but also the disease state, which will enhance clinical symptom follow-up, quality of life assessment, and research applications. Assessments of hypervigilance and visceral anxiety will complement these applications as these measures are both a PRO and an important moderator of symptom severity and quality of life.

摘要

患者报告结局(PRO)测量是评估患者与疾病和健康相关主观体验的重要工具。PRO在特定时间点测量一系列病症中的症状严重程度并评估治疗效果。尽管存在关注食管症状和食管过度警觉的PRO,但针对常见的良性食管疾病(如胃食管反流病(GERD)、嗜酸性粒细胞性食管炎(EoE)和贲门失弛缓症)的疾病特异性PRO有限。大多数GERD特异性PRO未能涵盖GERD的全部表现谱,而那些提供每日评估的PRO更适合研究。同样,许多EoE特异性PRO是为临床试验设计的。需要综合纳入EoE症状以及活动性炎症和纤维狭窄变化的内镜和组织学特征的工具。用于贲门失弛缓症的埃卡德特评分(Eckardt Score)的心理测量特性存在重大局限性,主要源于吞咽困难在评分中占主导地位。新的贲门失弛缓症特异性PRO试图通过捕捉细微的患者体验来克服这一问题。更广泛的症状PRO通常用于评估各种良性食管疾病的食管症状,包括一种评估食管过度警觉和症状特异性焦虑的PRO。未来的努力应集中在创建用户友好的PRO上,这些PRO不仅能全面评估临床表现,还能评估疾病状态,这将加强临床症状随访、生活质量评估和研究应用。对过度警觉和内脏焦虑的评估将补充这些应用,因为这些测量既是一种PRO,也是症状严重程度和生活质量的重要调节因素。

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