Pauling John D, Frech Tracy M, Domsic Robyn T, Hudson Marie
Royal National Hospital for Rheumatic Diseases, Bath; and Department of Pharmacy and Pharmacology, University of Bath, UK.
University of Utah and Salt Lake Regional Veterans Affair Medical Center, Salt Lake City, UT, USA.
Clin Exp Rheumatol. 2017 Sep-Oct;35 Suppl 106(4):184-192. Epub 2017 May 2.
The patient perspective captured using Patient-Reported Outcome (PRO) instruments provide insight into the patient condition not always captured by physician-derived assessment tools. Target patient population involvement is an essential component of PRO instrument development. We have reviewed the level of patient involvement in the development of PRO instruments used in the assessment of systemic sclerosis (SSc).
A comprehensive literature review was undertaken to identify studies reporting PRO instruments in SSc. Studies were assessed to establish whether the PRO instruments had been developed specifically for SSc or adopted from other disease areas. Studies reporting PRO instruments specific for SSc were scrutinised for evidence of target patient population involvement in the development of the instrument.
A total of 58 PRO instruments that have been used in SSc research were identified. Twelve (21%) of these were developed specifically for outcome assessment within SSc populations. Of these, 5 (42%) had not reported any patient involvement in the development phase of the instrument. Five SSc PRO instruments (42%) involved target patient population in the domain/item generation stage. Four (33%) of SSc PRO instruments had undertaken cognitive interviewing to ensure item wording adequately captured the intended conceptual framework.
The majority of PRO instruments used to assess SSc have not involved significant target patient involvement in their development. By involving patients in the development of novel PRO instruments in SSc, we can ensure such instruments adequately capture the experiences most relevant to our patients.
使用患者报告结局（PRO）工具所获取的患者视角，能为医师评估工具不一定能捕捉到的患者状况提供见解。目标患者群体的参与是PRO工具开发的重要组成部分。我们回顾了系统性硬化症（SSc）评估中使用的PRO工具开发过程中患者的参与程度。
进行全面的文献综述，以确定报告SSc中PRO工具的研究。评估这些研究，以确定PRO工具是否专门为SSc开发或从其他疾病领域采用。对报告专门针对SSc的PRO工具的研究进行审查，以寻找目标患者群体参与该工具开发的证据。
共确定了58种在SSc研究中使用的PRO工具。其中12种（21%）是专门为SSc人群的结局评估而开发的。在这些工具中，5种（42%）未报告在工具开发阶段有任何患者参与。5种SSc的PRO工具（42%）在领域/条目生成阶段让目标患者群体参与。4种（33%）SSc的PRO工具进行了认知访谈，以确保条目措辞能充分体现预期的概念框架。
用于评估SSc的大多数PRO工具在开发过程中并未让目标患者群体大量参与。通过让患者参与SSc新型PRO工具的开发，我们可以确保此类工具能充分捕捉与患者最相关的经历。
