O'Donovan Charles, Panthagani Jesse, Aiyegbusi Olalekan Lee, Liu Xiaoxuan, Bayliss Susan, Calvert Melanie, Pesudovs Konrad, Denniston Alastair, Moore David, Braithwaite Tasanee
School of Immunology and Microbiology, King's College London, London, England.
University Hospitals Birmingham, Birmingham, England.
J Ophthalmic Inflamm Infect. 2022 Sep 5;12(1):29. doi: 10.1186/s12348-022-00304-3.
Patient reported outcome measures (PROMs) capture impact of disease and treatment on quality of life, and have an emerging role in clinical trial outcome measurement. This study included a systematic review and quality appraisal of PROMs developed or validated for use in adults with uveitis or scleritis. We searched MEDLINE, EMBASE, PsycINFO, CINAHL and grey literature sources, to 5 November 2021. We used established quality criteria to grade each PROM instrument in multiple domains from A (high quality) to C (low quality), and assessed content development, validity, reliability and responsiveness. For instruments developed using classic test theory-based psychometric approaches, we assessed acceptability, item targeting and internal consistency. For instruments developed using Item Response Theory (IRT) (e.g. Rasch analysis), we assessed response categories, dimensionality, measurement precision, item fit statistics, differential item functioning and targeting. We identified and appraised four instruments applicable to certain uveitis types, but none for scleritis. Specifically, the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ), a 3-part PROM for Birdshot retinochoroiditis (Birdshot Disease & Medication Symptoms Questionnaire [BD&MSQ], the quality of life (QoL) impact of Birdshot Chorioretinopathy [QoL BCR], and the QoL impact of BCR medication [QoL Meds], the Kings Sarcoidosis Questionnaire (KSQ), and a PROM for cytomegalovirus retinitis. These instruments had limited coverage for these heterogeneous conditions, with a focus on very rare subtypes. Psychometric appraisal revealed considerable variability between instruments, limited content development, and only one developed using Item Response Theory. In conclusion, there are few validated PROMs for patients with uveitis and none for scleritis, and existing instruments have suboptimal psychometric performance. We articulate why we do not recommend their inclusion as clinical trial outcome measures for drug licensing purposes, and highlight an unmet need for PROMs applicable to uveitis and scleritis.
患者报告结局测量指标（PROMs）可反映疾病和治疗对生活质量的影响，在临床试验结局测量中发挥着越来越重要的作用。本研究对为葡萄膜炎或巩膜炎成人患者开发或验证的PROMs进行了系统评价和质量评估。我们检索了MEDLINE、EMBASE、PsycINFO、CINAHL和灰色文献来源，检索截止至2021年11月5日。我们使用既定的质量标准，在多个领域将每个PROM工具从A（高质量）到C（低质量）进行分级，并评估内容开发、效度、信度和反应度。对于使用基于经典测试理论的心理测量方法开发的工具，我们评估了可接受性、项目针对性和内部一致性。对于使用项目反应理论（IRT）（如拉施分析）开发的工具，我们评估了反应类别、维度、测量精度、项目拟合统计、项目功能差异和针对性。我们识别并评估了四种适用于某些葡萄膜炎类型的工具，但没有适用于巩膜炎的工具。具体来说，包括美国国立眼科研究所视觉功能问卷-25（NEI-VFQ），一种用于鸟枪弹样视网膜脉络膜炎的三部分PROM（鸟枪弹样疾病与药物症状问卷[BD&MSQ]、鸟枪弹样脉络膜视网膜炎的生活质量（QoL）影响[QoL BCR]以及BCR药物的QoL影响[QoL Meds]）、结节病问卷（KSQ）以及一种用于巨细胞病毒性视网膜炎的PROM。这些工具对这些异质性疾病的覆盖范围有限，主要关注非常罕见的亚型。心理测量评估显示，各工具之间存在很大差异，内容开发有限，且只有一种是使用项目反应理论开发的。总之，针对葡萄膜炎患者的经过验证的PROMs很少，针对巩膜炎患者的则没有，现有工具的心理测量性能欠佳。我们阐明了为何不建议将其作为药物许可目的的临床试验结局测量指标，并强调了对适用于葡萄膜炎和巩膜炎的PROMs的未满足需求。
