Statistics in Medicine

Article	Citations
Testing and correcting for weak and pleiotropic instruments in two‐sample multivariable Mendelian randomization	202
Minimum sample size for external validation of a clinical prediction model with a binary outcome	121
Sensitivity analysis for clinical trials with missing continuous outcome data using controlled multiple imputation: A practical guide	91
Extending inferences from a randomized trial to a new target population	89
Individual participant data meta‐analysis to examine interactions between treatment effect and participant‐level covariates: Statistical recommendations for conduct and planning	88
STRATOS guidance document on measurement error and misclassification of variables in observational epidemiology: Part 1—Basic theory and simple methods of adjustment	87
Graphical calibration curves and the integrated calibration index (ICI) for survival models	87
Evaluation of various estimators for standardized mean difference in meta‐analysis	76
Pleiotropy robust methods for multivariable Mendelian randomization	68
Minimum sample size for external validation of a clinical prediction model with a continuous outcome	47
Using propensity scores to estimate effects of treatment initiation decisions: State of the science	47
Formulating causal questions and principled statistical answers	45
STRATOS guidance document on measurement error and misclassification of variables in observational epidemiology: Part 2—More complex methods of adjustment and advanced topics	41
Assessing the performance of population adjustment methods for anchored indirect comparisons: A simulation study	39
Balancing vs modeling approaches to weighting in practice	37
Fine‐Gray subdistribution hazard models to simultaneously estimate the absolute risk of different event types: Cumulative total failure probability may exceed 1	36
Minimum sample size calculations for external validation of a clinical prediction model with a time‐to‐event outcome	33
A modified self‐controlled case series method for event‐dependent exposures and high event‐related mortality, with application to COVID‐19 vaccine safety	33
Estimating heterogeneous survival treatment effect in observational data using machine learning	32
Win odds: An adaptation of the win ratio to include ties	31
Analysis of time‐to‐event for observational studies: Guidance to the use of intensity models	29
Monitoring COVID‐19 contagion growth	28
Dismantling the Fragility Index: A demonstration of statistical reasoning	28
Handling missing predictor values when validating and applying a prediction model to new patients	27
Genome‐wide association study‐based deep learning for survival prediction	26

Bayesian workflow for disease transmission modeling in Stan	25
An overview and critique of the use of cumulative sum methods with surgical learning curve data	25
Introduction to computational causal inference using reproducible Stata, R, and Python code: A tutorial	25
Maintaining the validity of inference in small‐sample stepped wedge cluster randomized trials with binary outcomes when using generalized estimating equations	24
Propensity‐score‐based meta‐analytic predictive prior for incorporating real‐world and historical data	23
Nowcasting COVID‐19 incidence indicators during the Italian first outbreak	23
Matching with time‐dependent treatments: A review and look forward	22
A note on estimating the Cox‐Snell R2 from a reported C statistic (AUROC) to inform sample size calculations for developing a prediction model with a bin	22
Propensity score weighting for covariate adjustment in randomized clinical trials	21
A tutorial on individualized treatment effect prediction from randomized trials with a binary endpoint	21
A comparison of different methods to adjust survival curves for confounders	21
Extending the Mann‐Whitney‐Wilcoxon rank sum test to survey data for comparing mean ranks	20
TITE‐BOIN12: A Bayesian phase I/II trial design to find the optimal biological dose with late‐onset toxicity and efficacy	19
Penalized regression for left‐truncated and right‐censored survival data	19
Developing more generalizable prediction models from pooled studies and large clustered data sets	18
Sample size requirements for detecting treatment effect heterogeneity in cluster randomized trials	18
Comparing methods for estimating patient‐specific treatment effects in individual patient data meta‐analysis	18
One‐stage individual participant data meta‐analysis models for continuous and binary outcomes: Comparison of treatment coding options and estimation methods	18
Propensity score weighting for causal subgroup analysis	18
Bayesian semiparametric meta‐analytic‐predictive prior for historical control borrowing in clinical trials	18
Using fractional polynomials and restricted cubic splines to model non‐proportional hazards or time‐varying covariate effects in the Cox regression model	17
Sensitivity analysis of treatment effect to unmeasured confounding in observational studies with survival and competing risks outcomes	17
Transporting experimental results with entropy balancing	16
Confidence intervals of prediction accuracy measures for multivariable prediction models based on the bootstrap‐based optimism correction methods	16
A simplified stochastic EM algorithm for cure rate model with negative binomial competing risks: An application to breast cancer data	16
Propensity score methods for observational studies with clustered data: A review	16
Conflating marginal and conditional treatment effects: Comments on “Assessing the performance of population adjustment methods for anchored indirect comparisons: A simulation study”	15
Deep learning for survival outcomes	15
Bayesian model‐averaged meta‐analysis in medicine	15
Variable selection for high‐dimensional partly linear additive Cox model with application to Alzheimer's disease	15
Bayesian survival analysis with BUGS	15
Precision Bayesian phase I‐II dose‐finding based on utilities tailored to prognostic subgroups	15
Using electronic health records to identify candidates for human immunodeficiency virus pre‐exposure prophylaxis: An application of super learning to risk prediction when the outcome is rare	15
The statistical properties of RCTs and a proposal for shrinkage	15
Bayesian methods for the analysis of early‐phase oncology basket trials with information borrowing across cancer types	14
Differential expression of single‐cell RNA‐seq data using Tweedie models	14
Clinical prediction models to predict the risk of multiple binary outcomes: a comparison of approaches	14
Point estimation for adaptive trial designs I: A methodological review	14
Informing power and sample size calculations when using inverse probability of treatment weighting using the propensity score	14
A comparison of multiple imputation strategies for handling missing data in multi‐item scales: Guidance for longitudinal studies	14
Twenty years since Joinpoint 1.0: Two major enhancements, their justification, and impact	13
Mediation effect selection in high‐dimensional and compositional microbiome data	13
District‐level estimation of vaccination coverage: Discrete vs continuous spatial models	13
Sampling‐based estimation for massive survival data with additive hazards model	13
A two‐stage prediction model for heterogeneous effects of treatments	13
Generalizing randomized trial findings to a target population using complex survey population data	13
Extending the susceptible‐exposed‐infected‐removed (SEIR) model to handle the false negative rate and symptom‐based administration of COVID‐19 diagnostic tests: SEIR‐fansy	13
Accounting for unequal cluster sizes in designing cluster randomized trials to detect treatment effect heterogeneity	13
Multithreshold change plane model: Estimation theory and applications in subgroup identification	13
Deep reinforcement learning for personalized treatment recommendation	13

Raking and regression calibration: Methods to address bias from correlated covariate and time‐to‐event error	13
Avoiding bias in self‐controlled case series studies of coronavirus disease 2019	13
A two‐way flexible generalized gamma transformation cure rate model	13
Tumor heterogeneity estimation for radiomics in cancer	12
Statistical inference for decision curve analysis, with applications to cataract diagnosis	12
Uncertainty in the design stage of two‐stage Bayesian propensity score analysis	12
Bootstrap vs asymptotic variance estimation when using propensity score weighting with continuous and binary outcomes	12
On the properties of the toxicity index and its statistical efficiency	12
A Bayesian time‐to‐event pharmacokinetic model for phase I dose‐escalation trials with multiple schedules	12
Incorporating longitudinal biomarkers for dynamic risk prediction in the era of big data: A pseudo‐observation approach	12
Randomization‐based interval estimation in randomized clinical trials	12
Analysis of stepped wedge cluster randomized trials in the presence of a time‐varying treatment effect	12
Optimal diagnostic test allocation strategy during the COVID‐19 pandemic and beyond	12
Simultaneous modeling of Alzheimer's disease progression via multiple cognitive scales	12
Optimal multiwave sampling for regression modeling in two‐phase designs	11
A multiple imputation‐based sensitivity analysis approach for data subject to missing not at random	11
A tractable Bayesian joint model for longitudinal and survival data	11
Subgroup analysis in the heterogeneous Cox model	11
Adjusted logistic propensity weighting methods for population inference using nonprobability volunteer‐based epidemiologic cohorts	11
Bayesian inference using Hamiltonian Monte‐Carlo algorithm for nonlinear joint modeling in the context of cancer immunotherapy	11
Nonparametric machine learning for precision medicine with longitudinal clinical trials and Bayesian additive regression trees with mixed models	11
The impact of covariance priors on arm‐based Bayesian network meta‐analyses with binary outcomes	11
Prospective individual patient data meta‐analysis: Evaluating convalescent plasma for COVID‐19	11
Contamination: How much can an individually randomized trial tolerate?	11
On permutation tests for comparing restricted mean survival time with small sample from randomized trials	11
Estimation of seroconversion rates for infectious diseases: Effects of age and noise	11
An efficient variance estimator of AUC and its applications to binary classification	11
Point and interval estimation in two‐stage adaptive designs with time to event data and biomarker‐driven subpopulation selection	11
Selecting the model for multiple imputation of missing data: Just use an IC!	11
A practical introduction to Bayesian estimation of causal effects: Parametric and nonparametric approaches	11
Assessing vaccine durability in randomized trials following placebo crossover	10
Individual participant data meta‐analysis for external validation, recalibration, and updating of a flexible parametric prognostic model	10
A pilot design for observational studies: Using abundant data thoughtfully	10
Adaptive response‐dependent two‐phase designs: Some results on robustness and efficiency	10
A hidden Markov model for population‐level cervical cancer screening data	10
Confidence intervals for difference in proportions for matched pairs compatible with exact McNemar's or sign tests	10
Parameter clustering in Bayesian functional principal component analysis of neuroscientific data	10
A novel approach for propensity score matching and stratification for multiple treatments: Application to an electronic health record–derived study	10
The reciprocal Bayesian LASSO	10
Performance of variable and function selection methods for estimating the nonlinear health effects of correlated chemical mixtures: A simulation study	10
Generalized additive models to analyze nonlinear trends in biomedical longitudinal data using R: Beyond repeated measures ANOVA and linear mixed models	10
Design and analysis of three‐arm parallel cluster randomized trials with small numbers of clusters	10
Functional principal component analysis for longitudinal data with informative dropout	10
Statistical inference for missing data mechanisms	10
A new cure rate model with flexible competing causes with applications to melanoma and transplantation data	10
Score confidence intervals and sample sizes for stratified comparisons of binomial proportions	10
A Markov chain approach for ranking treatments in network meta‐analysis	10
A Bayesian modified Ising model for identifying spatially variable genes from spatial transcriptomics data	10
Bayesian learning of multiple directed networks from observational data	10
Geographically weighted generalized Farrington algorithm for rapid outbreak detection over short data accumulation periods	10
Optimizing subgroup selection in two‐stage adaptive enrichment and umbrella designs	10
Bayesian kernel machine regression‐causal mediation analysis	10
Propensity score trimming mitigates bias due to covariate measurement error in inverse probability of treatment weighted analyses: A plasmode simulation	10
Bayesian variable selection for understanding mixtures in environmental exposures	9
Binary genetic algorithm for optimal joinpoint detection: Application to cancer trend analysis	9
Estimating the sample variance from the sample size and range	9
Analytical methods for correlated data arising from multicenter hearing studies	9
Simulating the dynamics of atherosclerosis to the incidence of myocardial infarction, applied to the KORA population	9
When should matching be used in the design of cluster randomized trials?	9
Fusion designs and estimators for treatment effects	9
Robust estimation for linear panel data models	9
How large should the next study be? Predictive power and sample size requirements for replication studies	9
The effect of a constraint on the maximum number of controls matched to each treated subject on the performance of full matching on the propensity score when estimating risk differences	9
Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy	9
A multivariate statistical approach to predict COVID‐19 count data with epidemiological interpretation and uncertainty quantification	9
Target estimands for efficient decision making: Response to comments on “Assessing the performance of population adjustment methods for anchored indirect comparisons: A simulation study”	9
On a new piecewise regression model with cure rate: Diagnostics and application to medical data	9
Impact of discretization of the timeline for longitudinal causal inference methods	9
Estimation and modeling of the restricted mean time lost in the presence of competing risks	9
A forward search algorithm for detecting extreme study effects in network meta‐analysis	9
The length of the receiver operating characteristic curve and the two cutoff Youden index within a robust framework for discovery, evaluation, and cutoff estimation in biomarker studies involving impr	9
Parametric and nonparametric improvements in Bland and Altman's assessment of agreement method	9
The batched stepped wedge design: A design robust to delays in cluster recruitment	8
A joint model for multivariate longitudinal and survival data to discover the conversion to Alzheimer's disease	8
Flexible bivariate correlated count data regression	8
Measuring the performance of prediction models to personalize treatment choice	8
Pooling random forest and functional data analysis for biomedical signals supervised classification: Theory and application to electrocardiogram data	8
Bayesian variable selection with a pleiotropic loss function in Mendelian randomization	8
Sample size formula for a win ratio endpoint	8
Bayesian hierarchical models for high‐dimensional mediation analysis with coordinated selection of correlated mediators	8

Risk prediction models for discrete ordinal outcomes: Calibration and the impact of the proportional odds assumption	8
A bivariate logistic regression model based on latent variables	8
Imbalanced randomization in clinical trials	8
Accounting for publication bias using a bivariate trim and fill meta‐analysis procedure	8
Adaptively stacking ensembles for influenza forecasting	8
Bayesian latent factor on image regression with nonignorable missing data	8
Spline‐based accelerated failure time model	8
Reclaiming independence in spatial‐clustering datasets: A series of data‐driven spatial weights matrices	8
Progression models for repeated measures: Estimating novel treatment effects in progressive diseases	8
Early completion of phase I cancer clinical trials with Bayesian optimal interval design	8
Comparing Kaplan‐Meier curves with the probability of agreement	8
Regression with a right‐censored predictor using inverse probability weighting methods	8
A marginal estimate for the overall treatment effect on a survival outcome within the joint modeling framework	8
A Bayesian‐bandit adaptive design for N‐of‐1 clinical trials	8
Clustered spatio‐temporal varying coefficient regression model	8
A robust and unified framework for estimating heritability in twin studies using generalized estimating equations	8
Confounder selection strategies targeting stable treatment effect estimators	7
A puzzle of proportions: Two popular Bayesian tests can yield dramatically different conclusions	7
Improving coverage probabilities for parametric tolerance intervals via bootstrap calibration	7
Laplacian‐P‐splines for Bayesian inference in the mixture cure model	7
Flexible co‐data learning for high‐dimensional prediction	7
Smoothed time‐dependent receiver operating characteristic curve for right censored survival data	7
Household transmission of influenza A and B within a prospective cohort during the 2013‐2014 and 2014‐2015 seasons	7
A nonparametric method for value function guided subgroup identification via gradient tree boosting for censored survival data	7
Connecting Instrumental Variable methods for causal inference to the Estimand Framework	7
Scale mixture of skew‐normal linear mixed models with within‐subject serial dependence	7
Sequential knockoffs for continuous and categorical predictors: With application to a large psoriatic arthritis clinical trial pool	7
Deep learning for the dynamic prediction of multivariate longitudinal and survival data	7
On the robustness of latent class models for diagnostic testing with no gold standard	7
Target estimands for population‐adjusted indirect comparisons	7
Adaptive enrichment trials: What are the benefits?	7
Classification algorithm for high‐dimensional protein markers in time‐course data	7
Hierarchical multivariate directed acyclic graph autoregressive models for spatial diseases mapping	7
Bayesian analysis of multivariate linear mixed models with censored and intermittent missing responses	7
Maximum approximate Bernstein likelihood estimation in proportional hazard model for interval‐censored data	7
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Selection of variables for multivariable models: Opportunities and limitations in quantifying model stability by resampling	7
Multinomial logistic regression with missing outcome data: An application to cancer subtypes	7
An extension of the mixed‐effects growth model that considers between‐person differences in the within‐subject variance and the autocorrelation	7
Principal component analysis of hybrid functional and vector data	7
AIDS and COVID: A tale of two pandemics and the role of statisticians	7
Confidence interval estimation for treatment effects in cluster randomization trials based on ranks	7
uTPI: A utility‐based toxicity probability interval design for phase I/II dose‐finding trials	7
Using principal stratification in analysis of clinical trials	7
Multilevel modeling of spatially nested functional data: Spatiotemporal patterns of hospitalization rates in the US dialysis population	7
Window mean survival time	7
A tractable method to account for high‐dimensional nonignorable missing data in intensive longitudinal data	7
A simulation‐extrapolation approach for regression analysis of misclassified current status data with the additive hazards model	7
A multiparameter regression model for interval‐censored survival data	7
Bayesian adaptive N‐of‐1 trials for estimating population and individual treatment effects	7
To tolerate or to agree: A tutorial on tolerance intervals in method comparison studies with BivRegBLS R Package	6
Identifying density‐based local outliers in medical multivariate circular data	6
A Bayesian adaptive phase I/II platform trial design for pediatric immunotherapy trials	6
Unit information prior for adaptive information borrowing from multiple historical datasets	6
On the impact of residential history in the spatial analysis of diseases with a long latency period: A study of mesothelioma in Belgium	6
Optimal planning of adaptive two‐stage designs	6
A Bayesian approach for estimating typhoid fever incidence from large‐scale facility‐based passive surveillance data	6
Semiparametric regression analysis of partly interval‐censored failure time data with application to an AIDS clinical trial	6
Measuring association among censored antibody titer data	6
Propensity score methods for merging observational and experimental datasets	6
A tutorial on dealing with time‐varying eligibility for treatment: Comparing the risk of major bleeding with direct‐acting oral anticoagulants vs warfarin	6
A novel estimand to adjust for rescue treatment in randomized clinical trials	6
Sample size calculation for cluster randomization trials with a time‐to‐event endpoint	6
On the normalized power prior	6
Group testing in mediation analysis	6
Active learning for efficiently training emulators of computationally expensive mathematical models	6
Model diagnostics for censored regression via randomized survival probabilities	6
Restricted mean survival time for interval‐censored data	6
Integrated multiple mediation analysis: A robustness‐specificity trade‐off in causal structure	6
Joint analysis of mixed types of outcomes with latent variables	6
Six‐way decomposition of causal effects: Unifying mediation and mechanistic interaction	6
A unified approach to sample size and power determination for testing parameters in generalized linear and time‐to‐event regression models	6
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Assessing environmental epidemiology questions in practice with a causal inference pipeline: An investigation of the air pollution‐multiple sclerosis relapses relationship	6
A flexible nonlinear mixed effects model for HIV viral load rebound after treatment interruption	6
Gaussian graphical models with applications to omics analyses	6
Translating questions to estimands in randomized clinical trials with intercurrent events	6
Regression analysis for covariate‐adaptive randomization: A robust and efficient inference perspective	6
Computing the polytomous discrimination index	6
A comparison of parametric propensity score‐based methods for causal inference with multiple treatments and a binary outcome	6
Nonparametric covariate hypothesis tests for the cure rate in mixture cure models	6
Finite‐sample adjustments in variance estimators for clustered competing risks regression	6
A new conditional performance score for the evaluation of adaptive group sequential designs with sample size recalculation	6
Estimation of standard deviations and inverse‐variance weights from an observed range	6
Analysis of crossover designs with nonignorable dropout	6
Information content of stepped wedge designs with unequal cluster‐period sizes in linear mixed models: Informing incomplete designs	6
Profile‐likelihood Bayesian model averaging for two‐sample summary data Mendelian randomization in the presence of horizontal pleiotropy	6
Pitfalls of the concordance index for survival outcomes	5
Estimation of ascertainment bias and its effect on power in clinical trials with time‐to‐event outcomes	5
Robust estimation and variable selection for the accelerated failure time model	5
On the parameter estimation of Box‐Cox transformation cure model	5
A novel test by combining the maximum and minimum values among a large number of dependent Z‐scores with application to genome wide association study	5
Estimating the marginal hazard ratio by simultaneously using a set of propensity score models: A multiply robust approach	5
Sample size considerations for assessing treatment effect heterogeneity in randomized trials with heterogeneous intracluster correlations and variances	5
Regression analysis of arbitrarily censored survival data under the proportional odds model	5