A Systematic Review of Clinical Trials in Uveitis: Lessons Learned

Clinical trials in uveitis have led to the expansion of therapeutic options for the management of non-infectious uveitis. The purpose of this systematic review is to investigate why some clinical trials have yielded successful results and regulatory approval of new therapies, and some have not. A systematic literature search of the Pubmed/MEDLINE database and clinicaltrials.gov was performed from 2006 to 2021, according to the PRISMA guidelines. Phase III clinical trials of systemic and local therapies in adults with non-infectious intermediate, posterior, and panuveitis were included. A total of 79 clinical trials were collected from ClinicalTrials.gov and PubMed/MEDLINE database search. Based on the inclusion and exclusion criteria, 14 clinical trials were included. This review summarizes the study design, outcome measures, and results of recent phase III trials in non-infectious uveitis, in the interest of understanding limitations and rethinking new methods of defining endpoints in clinical trial design.

葡萄膜炎（uveitis）相关临床试验推动了非感染性葡萄膜炎治疗方案的拓展。本系统综述旨在探究为何部分临床试验可获得阳性结果并助力新型疗法获批监管许可，而另一部分则未能达成此目标。本研究遵循PRISMA指南（Preferred Reporting Items for Systematic Reviews and Meta-Analyses），于2006年至2021年间对PubMed/MEDLINE数据库及clinicaltrials.gov平台开展系统性文献检索。纳入针对罹患非感染性中间性、后性及全葡萄膜炎成年患者的全身与局部治疗Ⅲ期临床试验。通过检索ClinicalTrials.gov与PubMed/MEDLINE数据库，共检索到79项临床试验。经纳入与排除标准筛选后，最终纳入14项临床试验。本综述总结了近年来非感染性葡萄膜炎领域Ⅲ期临床试验的研究设计、结局指标与研究结果，以期明晰现有研究局限，并重新思考临床试验设计中结局定义的新范式。