年龄相关性黄斑变性临床试验的入组特征：一项横断面研究。
Enrolment characteristics in age-related macular degeneration clinical trials: a cross-sectional study.
作者信息
Tao Brendan K, Xie Jim S, Leung Victoria, Patel Milin, Xu Jeffrey, Lo Cody, Yan Peng, Nathoo Nawaaz, Lam Wai-Ching, Navajas Eduardo V, Muni Rajeev, Kohly Radha P
机构信息
Faculty of Medicine, The University of British Columbia, 317-2194 Health Sciences Mall, Vancouver, BC, V6T 1Z3, Canada.
Department of Ophthalmology and Vision Science, University of Toronto, Suite 400, 340 College Street, Toronto, ON, M5T 3A9, Canada.
出版信息
Eye (Lond). 2025 Apr;39(6):1153-1159. doi: 10.1038/s41433-024-03572-6. Epub 2024 Dec 27.
BACKGROUND/OBJECTIVES: To investigate demographic enrolment characteristics in age-related macular degeneration (AMD) trials.
SUBJECTS/METHODS: Clinicaltrials.gov was searched with "age-related macular degeneration" to identify RCTs with double, triple, or quadruple masking. Trial (e.g., study location, phase, masking, trial initiation year, and sponsor origin) and patient demographic data were collected. Sex-based AMD disease burdens were retrieved from the Global Burden of Disease database to calculate pooled female population-to-prevalence ratios (PPRs). Equitable trial enrolment was defined as PPR between 0.8-1.2. Demographic proportions were evaluated across trial characteristics using the Kruskal-Wallis test (alpha = 0.05) followed by post hoc comparisons. Secondary outcomes included absolute number of enrolled female, racial, and ethnic groups, and the association of trial characteristics with these demographics.
RESULTS
We included 106 trials (77,939 patients; 46.3% female), spanning 1990-2020. The pooled female PRR was 0.88 (95% confidence interval [CI]: 0.82, 0.94). PPR values were inconclusive studies with quadruple blinding; phase I, III, IV, or not applicable status; foreign (non-US) site; US-only and foreign-only sponsor; and an initiation year before 2010. There was no significant difference in PPR between sublevels of analysed trial characteristics. Of the 74 (69.8%) trials that adequately detailed race, White participants comprised the largest group (N = 57,917; 82% of total participants). Thirty-seven (34.9%) trials adequately detailed ethnicity. The absolute enrolment of race and ethnic groups did not generally exhibit significant difference between sublevels of analysed trial characteristics.
CONCLUSIONS
Female enrolment was commensurate to their disease burden across all trial characteristics. Race and ethnicity were under-reported. Future trials should prioritise equitable study enrolment strategies.
背景/目的：研究年龄相关性黄斑变性（AMD）试验中的人口统计学入组特征。
受试者/方法：在Clinicaltrials.gov上搜索“年龄相关性黄斑变性”，以确定采用双重、三重或四重盲法的随机对照试验（RCT）。收集试验（如研究地点、阶段、盲法、试验起始年份和申办方来源）和患者人口统计学数据。从全球疾病负担数据库中检索基于性别的AMD疾病负担，以计算合并的女性人口患病率比（PPR）。公平的试验入组定义为PPR在0.8至1.2之间。使用Kruskal-Wallis检验（α = 0.05）评估各试验特征的人口统计学比例，随后进行事后比较。次要结果包括入组女性的绝对数量、种族和民族群体，以及试验特征与这些人口统计学特征的关联。
结果
我们纳入了1990年至2020年期间的106项试验（77,939名患者；46.3%为女性）。合并的女性PPR为0.88（95%置信区间[CI]：0.82，0.94）。PPR值在采用四重盲法的不确定研究、I期、III期、IV期或不适用状态、国外（非美国）研究地点、仅美国和仅国外申办方以及2010年之前起始年份的研究中不明确。分析的试验特征子水平之间的PPR无显著差异。在74项（69.8%）充分详细说明种族的试验中，白人参与者占最大群体（N = 57,917；占总参与者的82%）。37项（34.9%）试验充分详细说明了民族。种族和民族群体的绝对入组人数在分析的试验特征子水平之间通常没有显著差异。
结论
在所有试验特征中，女性入组人数与其疾病负担相称。种族和民族的报告不足。未来的试验应优先考虑公平的研究入组策略。
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