Li, Xin Yung, Godwin Lin, Jianchang Zhu, Jian
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Statistics in medicine
Conditional power (CP) is a commonly used tool to inform interim decision-making in clinical trials, but the conventional approach using only primary endpoint data to calculate CP may not perform well when the primary endpoint requires a long follow-up period, or the treatment effect size changes during the trial. Several methods have been proposed...
Yu, Jih-Chang Huang, Yen-Tsung
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Statistics in medicine
Natural history of hepatitis B or C is comprised of multiple milestones such as liver cirrhosis and liver cancer. To fully characterize its natural course, semicompeting risks represent a common problem where liver cirrhosis and liver cancer are both of interest, but only the former may be censored by the latter. Copula, frailty and multistate mode...
Xiong, Ruoxuan Koenecke, Allison Powell, Michael Shen, Zhu Vogelstein, Joshua T Athey, Susan
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Statistics in medicine
We are interested in estimating the effect of a treatment applied to individuals at multiple sites, where data is stored locally for each site. Due to privacy constraints, individual-level data cannot be shared across sites; the sites may also have heterogeneous populations and treatment assignment mechanisms. Motivated by these considerations, we ...
Mamouris, Pavlos Nassiri, Vahid Verbeke, Geert Janssens, Arne Vaes, Bert Molenberghs, Geert
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Statistics in medicine
Imputation of longitudinal categorical covariates with several waves and many predictors is cumbersome in terms of implausible transitions, colinearity, and overfitting. We designed a simulation study with data obtained from a general practitioners' morbidity registry in Belgium for three waves, with smoking as the longitudinal covariate of interes...
Goyal, Ravi Carnegie, Nicole Slipher, Sally Turk, Philip Little, Susan J De Gruttola, Victor
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Statistics in medicine
To effectively mitigate the spread of communicable diseases, it is necessary to understand the interactions that enable disease transmission among individuals in a population; we refer to the set of these interactions as a contact network. The structure of the contact network can have profound effects on both the spread of infectious diseases and t...
Liang, Menglu Li, Zheng Li, Liang Chinchilli, Vernon M Zhang, Lijun Wang, Ming
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Statistics in medicine
In the field of cardiovascular disease, recurrent events such as stroke or myocardial infarction (MI) are often encountered, leading to an increase in the risk of death. Accurately evaluating the prognosis of patients and dynamically predicting the risk of death by considering the historical recurrent events can improve medical decisions and lead t...
de Jong, Valentijn M T Hoogland, Jeroen Moons, Karel G M Riley, Richard D Nguyen, Tri-Long Debray, Thomas P A
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Statistics in medicine
External validation of the discriminative ability of prediction models is of key importance. However, the interpretation of such evaluations is challenging, as the ability to discriminate depends on both the sample characteristics (ie, case-mix) and the generalizability of predictor coefficients, but most discrimination indices do not provide any i...
Tong, Guangyu Taljaard, Monica Li, Fan
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Statistics in medicine
An important consideration in the design and analysis of randomized trials is the need to account for outcome observations being positively correlated within groups or clusters. Two notable types of designs with this consideration are individually randomized group treatment trials and cluster randomized trials. While sample size methods for testing...
Ribaud, Mélina Gabriel, Edith Hughes, Joseph Soubeyrand, Samuel
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Statistics in medicine
Classical supervised methods like linear regression and decision trees are not completely adapted for identifying impacting factors on a response variable corresponding to zero-inflated proportion data (ZIPD) that are dependent, continuous and bounded. In this article we propose a within-block permutation-based methodology to identify factors (disc...
Kim, Sungduk Caporaso, Neil E Gu, Fangyi Klerman, Elizabeth B Albert, Paul S
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Statistics in medicine
Researchers in biology and medicine have increasingly focused on characterizing circadian rhythms and their potential impact on disease. Understanding circadian variation in metabolomics, the study of chemical processes involving metabolites may provide insight into important aspects of biological mechanism. Of scientific importance is developing a...