Patel D A, Sharda R, Hovis K L, Nichols E E, Sathe N, Penson D F, Feurer I D, McPheeters M L, Vaezi M F, Francis David O
Division of Gastroenterology, Hepatology and Nutrition.
Vanderbilt University School of Medicine.
Dis Esophagus. 2017 May 1;30(5):1-23. doi: 10.1093/dote/dow028.
Patient-reported outcome (PRO) measures are commonly used to capture patient experience with dysphagia and to evaluate treatment effectiveness. Inappropriate application can lead to distorted results in clinical studies. A systematic review of the literature on dysphagia-related PRO measures was performed to (1) identify all currently available measures and (2) to evaluate each for the presence of important measurement properties that would affect their applicability.
MEDLINE via the PubMed interface, the Cumulative Index of Nursing and Allied Health Literature, and the Health and Psychosocial Instrument database were searched using relevant vocabulary terms and key terms related to PRO measures and dysphagia. Three independent investigators performed abstract and full text reviews. Each study meeting criteria was evaluated using an 18-item checklist developed a priori that assessed multiple domains: (1) conceptual model, (2) content validity, (3) reliability, (4) construct validity, (6) scoring and interpretation, and (7) burden and presentation.
Of 4950 abstracts reviewed, a total of 34 dysphagia-related PRO measures (publication year 1987-2014) met criteria for extraction and analysis. Several PRO measures were of high quality (MADS for achalasia, SWAL-QOL and SSQ for oropharyngeal dysphagia, PROMIS-GI for general dysphagia, EORTC-QLQ-OG25 for esophageal cancer, ROMP-swallowing for Parkinson's Disease, DSQ-EoE for eosinophilic esophagitis, and SOAL for total laryngectomy-related dysphagia). In all, 17 met at least one criterion per domain. Thematic deficiencies in current measures were evident including: (1) direct patient involvement in content development, (2) empirically justified dimensionality, (3) demonstrable responsiveness to change, (4) plan for interpreting missing responses, and (5) literacy level assessment.
This is the first comprehensive systematic review assessing developmental properties of all available dysphagia-related PRO measures. We identified several instruments with robust measurement properties in multiple diseases including achalasia, oropharyngeal dysphagia, post-surgical dysphagia, esophageal cancer, and dysphagia related to neurological diseases. Findings herein can assist clinicians and researchers in making more informed decisions in selecting the most fundamentally sound PRO measure for a given clinical, research, or quality initiative.
患者报告结局(PRO)测量通常用于了解患者吞咽困难的体验并评估治疗效果。应用不当可能导致临床研究结果失真。对与吞咽困难相关的PRO测量的文献进行系统综述,以(1)识别所有当前可用的测量方法,以及(2)评估每种方法是否具有会影响其适用性的重要测量属性。
通过PubMed界面检索MEDLINE、护理及相关健康文献累积索引以及健康和心理社会测量工具数据库,使用与PRO测量和吞咽困难相关的相关词汇和关键词。三位独立研究人员进行摘要和全文审查。每项符合标准的研究均使用事先制定的18项清单进行评估,该清单评估多个领域:(1)概念模型,(2)内容效度,(3)信度,(4)结构效度,(6)评分与解释,以及(7)负担与呈现。
在审查的4950篇摘要中,共有34项与吞咽困难相关的PRO测量方法(发表年份1987 - 2014年)符合提取和分析标准。几种PRO测量方法质量较高(贲门失弛缓症的MADS、口咽吞咽困难的SWAL - QOL和SSQ、一般吞咽困难的PROMIS - GI、食管癌的EORTC - QLQ - OG25、帕金森病的ROMP - 吞咽、嗜酸性食管炎的DSQ - EoE以及全喉切除相关吞咽困难的SOAL)。总体而言,17项至少在每个领域符合一项标准。当前测量方法存在明显的主题缺陷,包括:(1)患者直接参与内容开发,(2)基于经验的合理维度,(3)对变化的可证明的反应性,(4)解释缺失反应的计划,以及(5)识字水平评估。
这是首次全面系统综述评估所有可用的与吞咽困难相关的PRO测量方法的发展属性。我们确定了几种在多种疾病(包括贲门失弛缓症、口咽吞咽困难、手术后吞咽困难、食管癌以及与神经系统疾病相关的吞咽困难)中具有强大测量属性的工具。本文的研究结果可帮助临床医生和研究人员在为特定临床、研究或质量计划选择最根本合理的PRO测量方法时做出更明智的决策。