A quantitative approach to the spread of variance in translational research using Monte Carlo simulation.
A quantitative approach to the spread of variance in translational research using Monte Carlo simulation.
复制标题
DOI：
10.1038/s41598-022-09921-3
复制
发表时间：
2022-04-15
影响因子：
4.6
通讯作者：
Levin, Leonard A.
中科院分区：
文献类型：
--
作者：
Cukurova, Feyza;Gustavson, Britta P.;Griborio-Guzman, Andres G.;Levin, Leonard A.
将有前途的临床前研究转化为成功的试验往往会失败。一个促成因素是“公主与豌豆”问题，这是指随着研究过渡到更复杂的系统，最初显著的效应大小如何消散。这项工作的目的是量化扩散变异性对样本量要求的影响。通过Monte Carlo模拟进行样本量估计。为了模拟从临床前研究进展到临床研究的过程，使用了具有可修改输入参数变异性的嵌套S形剂量-反应转换。结果表明，与标准计算相比，向剂量-反应参数添加变异性显著增加了样本量要求。增加连续研究的数量进一步增加了样本量。这些结果定量地证明了如何在翻译研究中传播的变异性，这是不典型的占，可以导致急剧增加所需的样本量，以保持所需的研究力量。
The translation of promising preclinical research into successful trials often fails. One contributing factor is the “Princess and the Pea” problem, which refers to how an initially significant effect size dissipates as research transitions to more complex systems. This work aimed to quantify the effects of spreading variability on sample size requirements. Sample size estimates were performed by Monte Carlo simulation. To simulate the process of progressing from preclinical to clinical studies, nested sigmoidal dose–response transformations with modifiable input parameter variability were used. The results demonstrated that adding variabilty to the dose–response parameters substantially increases sample size requirements compared to standared calculations. Increasing the number of consecutive studies further increases the sample size. These results quantitatively demonstrate how the spread of variability in translational research, which is not typically accounted for, can result in drastic increases in the sample size required to maintain a desired study power.
登录
查看更多内容
影响因子：
7.4
作者：
Lim, Mark David;Dickherber, Anthony;Compton, Carolyn C.
通讯作者：
Compton, Carolyn C.
影响因子：
1.1
作者：
Chow, Shein-Chung;Shao, Jun;Wang, Hansheng
通讯作者：
Wang, Hansheng
影响因子：
8.8
作者：
Kreznar JH;Keller MP;Traeger LL;Rabaglia ME;Schueler KL;Stapleton DS;Zhao W;Vivas EI;Yandell BS;Broman AT;Hagenbuch B;Attie AD;Rey FE
通讯作者：
Rey FE
影响因子：
4
作者：
Ergorul, Ceren;Levin, Leonard A.
通讯作者：
Levin, Leonard A.
DOI：
10.1073/pnas.96.17.9944
发表时间：
1999-08-17
影响因子：
11.1
作者：
Neufeld, AH;Sawada, A;Becker, B
通讯作者：
Becker, B