Tadić V, Rahi J S
Life Course Epidemiology and Biostatistics Section, Population, Policy and Practice Programme, UCL Great Ormond Street Institute of Child Health, London, UK.
Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Eye (Lond). 2017 Apr;31(4):511-518. doi: 10.1038/eye.2016.316. Epub 2017 Jan 13.
The purpose of this article is to summarise methodological challenges and opportunities in the development and application of patient reported outcome measures (PROMs) for the rare and complex population of children with visually impairing disorders. Following a literature review on development and application of PROMs in children in general, including those with disabilities and or/chronic condition, we identified and discuss here 5 key issues that are specific to children with visual impairment: (1) the conflation between theoretically distinct vision-related constructs and outcomes, (2) the importance of developmentally appropriate approaches to design and application of PROMs, (3) feasibility of standard questionnaire formats and administration for children with different levels of visual impairment, (4) feasibility and nature of self-reporting by visually impaired children, and (5) epidemiological, statistical and ethical considerations. There is an established need for vision-specific age-appropriate PROMs for use in paediatric ophthalmology, but there are significant practical and methodological challenges in developing and applying appropriate measures. Further understanding of the characteristics and needs of visually impaired children as questionnaire respondents is necessary for development of quality PROMs and their meaningful application in clinical practice and research.
本文旨在总结针对患有视力障碍疾病的罕见且复杂儿童群体，在患者报告结局测量指标（PROMs）的开发与应用过程中所面临的方法学挑战及机遇。在对PROMs在一般儿童（包括残疾儿童和/或慢性病儿童）中的开发与应用进行文献综述之后，我们在此识别并讨论了视力障碍儿童所特有的5个关键问题：（1）理论上不同的与视力相关的概念和结局之间的混淆；（2）采用适合发育阶段的方法来设计和应用PROMs的重要性；（3）标准问卷格式以及针对不同视力障碍程度儿童进行问卷管理的可行性；（4）视力障碍儿童自我报告的可行性及性质；（5）流行病学、统计学和伦理学方面的考量。在儿科眼科领域，确实需要针对特定视力情况且适合相应年龄段的PROMs，但在开发和应用合适的测量指标方面存在重大的实践和方法学挑战。为了开发高质量的PROMs并使其在临床实践和研究中得到有意义的应用，有必要进一步了解视力障碍儿童作为问卷受访者的特征和需求。
