• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

比较儿科与成人随机试验中的不良事件参数：一项荟萃流行病学实证评估。

Comparison of nuisance parameters in pediatric versus adult randomized trials: a meta-epidemiologic empirical evaluation.

机构信息

Department of Pediatrics, Alberta Research Centre for Health Evidence, University of Alberta, Edmonton, Canada.

Department of Biostatistics, Julius Centre for Health Sciences and Primary Care, University Medical Centre, Utrecht, Netherlands.

出版信息

BMC Med Res Methodol. 2018 Jan 10;18(1):7. doi: 10.1186/s12874-017-0456-8.

DOI:10.1186/s12874-017-0456-8
PMID:29321002
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5763521/
Abstract

BACKGROUND

We wished to compare the nuisance parameters of pediatric vs. adult randomized-trials (RCTs) and determine if the latter can be used in sample size computations of the former.

METHODS

In this meta-epidemiologic empirical evaluation we examined meta-analyses from the Cochrane Database of Systematic-Reviews, with at least one pediatric-RCT and at least one adult-RCT. Within each meta-analysis of binary efficacy-outcomes, we calculated the pooled-control-group event-rate (CER) across separately all pediatric and adult-trials, using random-effect models and subsequently calculated the control-group event-rate risk-ratio (CER-RR) of the pooled-pediatric-CERs vs. adult-CERs. Within each meta-analysis with continuous outcomes we calculated the pooled-control-group effect standard deviation (CE-SD) across separately all pediatric and adult-trials and subsequently calculated the CE-SD-ratio of the pooled-pediatric-CE-SDs vs. adult-CE-SDs. We then calculated across all meta-analyses the pooled-CER-RRs and pooled-CE-SD-ratios (primary endpoints) and the pooled-magnitude of effect-sizes of CER-RRs and CE-SD-ratios using REMs. A ratio < 1 indicates that pediatric trials have smaller nuisance parameters than adult trials.

RESULTS

We analyzed 208 meta-analyses (135 for binary-outcomes, 73 for continuous-outcomes). For binary outcomes, pediatric-RCTs had on average 10% smaller CERs than adult-RCTs (summary-CE-RR: 0.90; 95% CI: 0.83, 0.98). For mortality outcomes the summary-CE-RR was 0.48 (95% CIs: 0.31, 0.74). For continuous outcomes, pediatric-RCTs had on average 26% smaller CE-SDs than adult-RCTs (summary-CE-SD-ratio: 0.74).

CONCLUSIONS

Clinically relevant differences in nuisance parameters between pediatric and adult trials were detected. These differences have implications for design of future studies. Extrapolation of nuisance parameters for sample-sizes calculations from adult-trials to pediatric-trials should be cautiously done.

摘要

背景

我们希望比较儿科和成人随机对照试验（RCT）的干扰参数，并确定后者是否可以用于前者的样本量计算。

方法

在这项荟萃流行病学实证评估中，我们检查了 Cochrane 系统评价数据库中的荟萃分析，其中至少有一项儿科 RCT 和至少一项成人 RCT。在每个二元疗效结局的荟萃分析中，我们使用随机效应模型分别计算了所有儿科和成人试验的汇总对照组事件率（CER），并随后计算了汇总儿科 CER 与成人 CER 的对照组事件率风险比（CER-RR）。在每个连续结局的荟萃分析中，我们分别计算了所有儿科和成人试验的汇总对照组效应标准差（CE-SD），并随后计算了汇总儿科 CE-SD 与成人 CE-SD 的 CE-SD 比值。然后，我们使用 REM 计算了所有荟萃分析的汇总 CER-RR 和汇总 CE-SD 比值（主要终点）以及 CER-RR 和 CE-SD 比值的效应大小的汇总幅度。比值<1 表示儿科试验的干扰参数小于成人试验。

结果

我们分析了 208 项荟萃分析（135 项用于二元结局，73 项用于连续结局）。对于二元结局，儿科 RCT 的 CER 平均比成人 RCT 小 10%（汇总 CE-RR：0.90；95%CI：0.83，0.98）。对于死亡率结局，汇总 CE-RR 为 0.48（95%CI：0.31，0.74）。对于连续结局，儿科 RCT 的 CE-SD 平均比成人 RCT 小 26%（汇总 CE-SD 比值：0.74）。

结论

在儿科和成人试验的干扰参数之间检测到了临床相关的差异。这些差异对未来研究的设计有影响。从成人试验外推到儿科试验的样本量计算的干扰参数应谨慎进行。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/48ff9144dfbc/12874_2017_456_Fig6_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/a3ee420e19a1/12874_2017_456_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/d5b20789f2c8/12874_2017_456_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/3310b2b16570/12874_2017_456_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/b5842b19cfbe/12874_2017_456_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/703f438aea45/12874_2017_456_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/48ff9144dfbc/12874_2017_456_Fig6_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/a3ee420e19a1/12874_2017_456_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/d5b20789f2c8/12874_2017_456_Fig2_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/3310b2b16570/12874_2017_456_Fig3_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/b5842b19cfbe/12874_2017_456_Fig4_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/703f438aea45/12874_2017_456_Fig5_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d6b0/5763521/48ff9144dfbc/12874_2017_456_Fig6_HTML.jpg

相似文献

1
Comparison of nuisance parameters in pediatric versus adult randomized trials: a meta-epidemiologic empirical evaluation.比较儿科与成人随机试验中的不良事件参数：一项荟萃流行病学实证评估。
BMC Med Res Methodol. 2018 Jan 10;18(1):7. doi: 10.1186/s12874-017-0456-8.
2
Sensitivity subgroup analysis based on single-center vs. multi-center trial status when interpreting meta-analyses pooled estimates: the logical way forward.在解释荟萃分析合并估计值时，基于单中心与多中心试验状态的敏感性亚组分析：未来的合理方法。
J Clin Epidemiol. 2016 Jun;74:80-92. doi: 10.1016/j.jclinepi.2015.08.027. Epub 2015 Nov 17.
3
Comparative evidence on harms in pediatric randomized clinical trials from less developed versus more developed countries is limited.与发达国家相比，来自欠发达国家的儿科随机临床试验中的危害比较证据有限。
J Clin Epidemiol. 2018 Mar;95:63-72. doi: 10.1016/j.jclinepi.2017.11.016. Epub 2017 Nov 28.
4
Sample Size Calculation: Inaccurate A Priori Assumptions for Nuisance Parameters Can Greatly Affect the Power of a Randomized Controlled Trial.样本量计算：对干扰参数的先验假设不准确会极大地影响随机对照试验的效能。
PLoS One. 2015 Jul 14;10(7):e0132578. doi: 10.1371/journal.pone.0132578. eCollection 2015.
5
6
Comparison of large versus smaller randomized trials for mental health-related interventions.大型与小型心理健康相关干预随机试验的比较。
Am J Psychiatry. 2005 Mar;162(3):578-84. doi: 10.1176/appi.ajp.162.3.578.
7
Single-center trials show larger treatment effects than multicenter trials: evidence from a meta-epidemiologic study.单中心试验比多中心试验显示出更大的治疗效果：来自荟萃流行病学研究的证据。
Ann Intern Med. 2011 Jul 5;155(1):39-51. doi: 10.7326/0003-4819-155-1-201107050-00006.
8
9
Pediatric Tui Na for acute diarrhea in children under 5 years old: A systematic review and meta-analysis of randomized clinical trials.小儿推拿治疗 5 岁以下儿童急性腹泻：随机临床试验的系统评价和荟萃分析。
Complement Ther Med. 2018 Dec;41:10-22. doi: 10.1016/j.ctim.2018.08.011. Epub 2018 Aug 30.
10
Pre-operative endometrial thinning agents before endometrial destruction for heavy menstrual bleeding.对于月经过多患者，在进行子宫内膜破坏术前使用的术前子宫内膜减薄剂。
Cochrane Database Syst Rev. 2013 Nov 15;2013(11):CD010241. doi: 10.1002/14651858.CD010241.pub2.

引用本文的文献

1
The Constrained Disorder Principle Overcomes the Challenges of Methods for Assessing Uncertainty in Biological Systems.约束无序原则克服了生物系统不确定性评估方法的挑战。
J Pers Med. 2024 Dec 28;15(1):10. doi: 10.3390/jpm15010010.
2
Off-label use of medicines in South Africa: a review.南非的药品标签外使用：综述。
Orphanet J Rare Dis. 2024 Nov 29;19(1):448. doi: 10.1186/s13023-024-03476-4.
3
Off-Label and Unlicenced Medicine Use among Hospitalised Children in South Africa: Practice and Policy Implications.南非住院儿童的超说明书用药和未获许可药物使用：实践与政策影响

本文引用的文献

1
Meta-epidemiological study: a step-by-step approach by using R.Meta流行病学研究：使用R语言的逐步方法。
J Evid Based Med. 2016 May;9(2):91-97. doi: 10.1111/jebm.12191.
2
Sample size calculations in pediatric clinical trials conducted in an ICU: a systematic review.在重症监护病房进行的儿科临床试验中的样本量计算：一项系统评价。
Trials. 2014 Jul 8;15:274. doi: 10.1186/1745-6215-15-274.
3
Safety of medical interventions in children versus adults.儿童与成人医疗干预的安全性比较。
Pharmacy (Basel). 2023 Nov 9;11(6):174. doi: 10.3390/pharmacy11060174.
4
Age-treatment subgroup analyses in Cochrane intervention reviews: a meta-epidemiological study.Cochrane 干预评价中年龄-处理亚组分析：一项荟萃流行病学研究。
BMC Med. 2019 Oct 21;17(1):188. doi: 10.1186/s12916-019-1420-8.
Pediatrics. 2014 Mar;133(3):e666-73. doi: 10.1542/peds.2013-3128. Epub 2014 Feb 24.
4
Are sample sizes clear and justified in RCTs published in dental journals?牙科期刊上发表的随机对照试验中的样本量是否明确且合理？
PLoS One. 2014 Jan 21;9(1):e85949. doi: 10.1371/journal.pone.0085949. eCollection 2014.
5
Standard 4: determining adequate sample sizes.标准4：确定足够的样本量。
Pediatrics. 2012 Jun;129 Suppl 3:S138-45. doi: 10.1542/peds.2012-0055G.
6
Comparative effectiveness of medical interventions in adults versus children.成人与儿童医疗干预措施的比较效果。
J Pediatr. 2010 Aug;157(2):322-330.e17. doi: 10.1016/j.jpeds.2010.02.011.
7
Bayesian design using adult data to augment pediatric trials.利用成人数据增强儿科试验的贝叶斯设计。
Clin Trials. 2009 Aug;6(4):297-304. doi: 10.1177/1740774509339238.
8
A power primer.强力底漆。
Psychol Bull. 1992 Jul;112(1):155-9. doi: 10.1037//0033-2909.112.1.155.
9
Pitfalls in the design and analysis of paediatric clinical trials: a case of a 'failed' multi-centre study, and potential solutions.儿科临床试验设计与分析中的陷阱：以一项“失败”的多中心研究为例及潜在解决方案
Acta Paediatr. 2009 Feb;98(2):385-91. doi: 10.1111/j.1651-2227.2008.01048.x. Epub 2008 Sep 24.
10
Children are not just small adults: the urgent need for high-quality trial evidence in children.儿童并非只是缩小版的成人：迫切需要高质量的儿童试验证据。
PLoS Med. 2008 Aug 12;5(8):e172. doi: 10.1371/journal.pmed.0050172.