基于循证医学构建白内障患者报告结局条目池

Development of cataract-specific item pool to measure patient-reported outcomes

目的：研制基于循证医学方法的白内障患者报告结局（PROs）条目池，为合理评估白内障PROs奠定了基础，并为其他类似研究提供参考。方法循证医学研究。遵循国际PROs测量工具研制流程构建白内障PROs条目池。首先建立条目池理论框架，再通过计算机检索美国国立图书馆医学文献检索系统（PubMed）、荷兰医学文摘数据库（Embase）、中国知网（CNKI）、万方期刊论文数据库（WanFang Data）、维普中文科技期刊全文数据库（VIP）、中国生物医学文献数据库（CBM）等数据库，纳入所有白内障PROs测量工具，进一步按照条目初筛、跨文化调试、患者访谈、条目分类及再次筛选、专家评审、确立条目主干及选项、人群验证等步骤构建白内障PROs条目池。结果条目池理论框架由视觉症状、视觉相关活动受限和精神心理健康等3个维度构成。通过文献检索后纳入19个PROs量表，条目初筛后纳入249个条目，患者访谈后保留204个条目，条目分类及再次筛选后保留118个条目，专家评审后保留94个条目，人群验证后形成的白内障条目池最终版本包括94个条目，其中视觉症状维度28个条目、视觉相关活动受限维度41个条目、精神心理健康维度25个条目，所有条目选项个数均为4个。结论本研究基于PROs循证医学方法，规范地构建了包含视觉症状、视觉相关活动受限和精神心理健康这三大维度的白内障PROs条目池，具有语言通俗易懂、内容全面并能反应视功能损害等特点，改善了目前眼科领域PROs测量工具的缺陷，为白内障PROs条目池构建的规范化提供了参考。

Objective Based on the principles of evidence-based medicine, the present study aimed to develop a cataract-specific item pool for the assessment of quality of life （ QoL ） in ophthalmic patients and the efficacy of specific therapy. Methods The item pool for patient reported outcomes （ PROs ） of cataract was established based on the guideline of the international PROs development process. Databases including PubMed, Embase, CNKI, WanFang Data, VIP, CBM were thoroughly searched. The items pool of PROs was established using the following steps： Confirm conceptual framework, identify extant items in cataract-specific instruments and the qualitative literature, select items and cross-cultural adaptation, focus groups and semi-structured interviews, item classification and selection, identify items stem and response options, expert review and revision of items, cognitive interviews and develop final version of instrument. Results The conceptual framework of item pool consists of vision-related activity limitation （VRAL） domain, vision symptoms domain and emotional well-being domain. After manual scanning for cataract-specific instruments, 19 questionnaires were extracted for the initial item pool development. After the original identification of items, focus groups and semi-structured interviews, item classification and selection, expert review and cognitive interviews, the number of items were 249, 204, 118 and 94, respectively. After cognitive interviews, all items were sorted into VRAL domain, vision symptoms domain and emotional well-being domain, which contained 41, 28 and 25 items, respectively. Total items utilized four response options. Conclusion Based on the principles of evidence-based medicine for PROs, the final item pool contained majority of the items under VRAL, vision symptoms and emotional well-being domains. Consequently, the language of items under each domain was simplified for interpretation by the patients, and possessed perfect content validity. This cataract-specific item pool has corrected the defects of PROs instruments in ophthalmology , and provided a formal reference for establishing item pools.