Department of Pediatrics, Stanford University School of Medicine, Stanford, California.
University of Washington School of Medicine, Seattle, Washington.
Pediatrics. 2022 Apr 1;149(4). doi: 10.1542/peds.2021-052557.
Unique ethical, epidemiological, and economic factors are barriers to performing research in children. The landscape of pediatric clinical trials, including drivers of completion and timely dissemination of results, is not well understood. We aimed to characterize the prevalence of and factors associated with early discontinuation, results reporting, and publication of pediatric clinical trials registered at ClinicalTrials.gov.
Cross-sectional analysis of clinical trials enrolling participants <18 years old registered at ClinicalTrials.gov from October 2007 to March 2020. Multivariable logistic regressions were performed to assess the association between trial characteristics and primary outcomes. Publication data were obtained through PubMed, ClinicalTrials.gov, Embase, and Scopus.
Overall, 11.1% trials were stopped early, with recruitment failure being the predominant reason for discontinuation. Only 23.5% of completed trials reported results, and 38.8% were published within 3 years of completion. Rates of discontinuation and publication significantly improved over the study period. Among funding sources, government-sponsored trials (adjusted odds ratio [aOR], 0.72; 95% CI, 0.47-0.97) and academic trials (aOR, 0.64; 95% CI, 0.50-0.82) had lower odds of discontinuation compared with industry trials and were more likely to be published (government: aOR, 1.94 [95% CI, 1.52-2.48] academic: aOR, 1.61 [95% CI, 1.35-1.92). Academic trial investigators were the least likely to report results (aOR, 0.34; 95% CI, 0.31-0.52).
Early discontinuation and nonreporting/nonpublication of findings remain common in registered pediatric clinical trials and were associated with funding source and other trial features. Targeted efforts are needed to support trial completion and timely results dissemination toward strengthening evidence-based pediatric medicine.
在儿童中开展研究存在独特的伦理、流行病学和经济方面的障碍。儿科临床试验的情况，包括完成情况和及时传播结果的驱动因素，尚未得到充分了解。我们旨在描述在 ClinicalTrials.gov 注册的儿科临床试验中早期终止、结果报告和发表的发生率和相关因素。
对 2007 年 10 月至 2020 年 3 月在 ClinicalTrials.gov 注册的招募参与者年龄<18 岁的临床试验进行横断面分析。采用多变量逻辑回归评估试验特征与主要结局之间的关联。通过 PubMed、ClinicalTrials.gov、Embase 和 Scopus 获得发表数据。
总体而言，有 11.1%的试验提前终止，招募失败是终止的主要原因。只有 23.5%的完成试验报告了结果，38.8%在完成后 3 年内发表。研究期间，终止和发表的比例显著提高。在资金来源方面，与工业试验相比，政府资助的试验（调整后的优势比[OR]，0.72；95%置信区间[CI]，0.47-0.97）和学术试验（aOR，0.64；95%CI，0.50-0.82）终止的可能性较低，更有可能发表（政府：aOR，1.94[95%CI，1.52-2.48]；学术：aOR，1.61[95%CI，1.35-1.92）。学术试验的研究者报告结果的可能性最低（aOR，0.34；95%CI，0.31-0.52）。
在注册的儿科临床试验中，早期终止和不报告/不发表研究结果仍然很常见，与资金来源和其他试验特征有关。需要有针对性地努力支持试验完成和及时传播结果，以加强循证儿科学。
