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covariate_adjustment.bib
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covariate_adjustment.bib
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%%% OUR REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{VanLancker2022,
doi = {10.48550/ARXIV.2201.12921},
url = {https://arxiv.org/abs/2201.12921},
author = {Van Lancker, Kelly and Betz, Joshua and Rosenblum, Michael},
keywords = {Methodology (stat.ME), FOS: Computer and information sciences, FOS: Computer and information sciences},
title = {Combining Covariate Adjustment with Group Sequential, Information Adaptive Designs to Improve Randomized Trial Efficiency},
journal={arXiv preprint arXiv:1409.0473},
publisher = {arXiv},
year = {2022},
copyright = {Creative Commons Attribution 4.0 International}
}
%%% R PACKAGE REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@Manual{boot_package,
title = {boot: Bootstrap R (S-Plus) Functions},
author = {Angelo Canty and B. D. Ripley},
year = {2022},
note = {R package version 1.3-28.1},
url = {https://cran.r-project.org/web/packages/boot/}
}
@Manual{margins_package,
title = {margins: Marginal Effects for Model Objects},
author = {Thomas J. Leeper},
year = {2021},
note = {R package version 0.3.26},
url = {https://cran.r-project.org/web/packages/margins}
}
@Manual{rpact_package,
title = {rpact: Confirmatory Adaptive Clinical Trial Design and Analysis},
author = {Gernot Wassmer and Friedrich Pahlke},
year = {2023},
note = {R package version 3.4.0},
url = {https://CRAN.R-project.org/web/packages/rpact},
}
@article{Zeileis2006,
doi = {10.18637/jss.v016.i09},
url = {https://doi.org/10.18637/jss.v016.i09},
year = {2006},
publisher = {Foundation for Open Access Statistic},
volume = {16},
number = {9},
author = {Achim Zeileis},
title = {Object-Oriented Computation of Sandwich Estimators},
journal = {Journal of Statistical Software}
}
%%% CLINICAL TRIALS REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Assmann2000,
doi = {10.1016/s0140-6736(00)02039-0},
url = {https://doi.org/10.1016/s0140-6736(00)02039-0},
year = {2000},
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publisher = {Elsevier {BV}},
volume = {355},
number = {9209},
pages = {1064--1069},
author = {Susan F Assmann and Stuart J Pocock and Laura E Enos and Linda E Kasten},
title = {Subgroup analysis and other (mis)uses of baseline data in clinical trials},
journal = {The Lancet}
}
@article{Schulz2010,
doi = {10.1136/bmj.c332},
url = {https://doi.org/10.1136/bmj.c332},
year = {2010},
month = mar,
publisher = {{BMJ}},
volume = {340},
number = {mar23 1},
pages = {c332--c332},
author = {K. F Schulz and D. G Altman and D. Moher and},
title = {{CONSORT} 2010 Statement: updated guidelines for reporting parallel group randomised trials},
journal = {{BMJ}}
}
@article{Gamble2017,
doi = {10.1001/jama.2017.18556},
url = {https://doi.org/10.1001/jama.2017.18556},
year = {2017},
month = dec,
publisher = {American Medical Association ({AMA})},
volume = {318},
number = {23},
pages = {2337},
author = {Carrol Gamble and Ashma Krishan and Deborah Stocken and Steff Lewis and Edmund Juszczak and Caroline Dor{\'{e}} and Paula R. Williamson and Douglas G. Altman and Alan Montgomery and Pilar Lim and Jesse Berlin and Stephen Senn and Simon Day and Yolanda Barbachano and Elizabeth Loder},
title = {Guidelines for the Content of Statistical Analysis Plans in Clinical Trials},
journal = {{JAMA}}
}
%%% INFORMATION MONITORING REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Mehta2001,
title = {Flexible Sample Size Considerations Using Information-Based Interim Monitoring},
volume = {35},
ISSN = {2164-9200},
url = {http://dx.doi.org/10.1177/009286150103500407},
DOI = {10.1177/009286150103500407},
number = {4},
journal = {Drug Information Journal},
publisher = {Springer Science and Business Media LLC},
author = {Mehta, Cyrus R. and Tsiatis, Anastasios A.},
year = {2001},
month = oct,
pages = {1095–1112}
}
%%% GROUP SEQUENTIAL DESIGNS REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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doi = {10.1201/9780367805326},
url = {https://doi.org/10.1201/9780367805326},
year = {1999},
month = sep,
publisher = {Chapman and Hall/{CRC}},
author = {Christopher Jennison and Bruce W. Turnbull},
title = {Group Sequential Methods with Applications to Clinical Trials}
}
@article{Lakens2021,
title = {Group Sequential Designs: A Tutorial},
url = {http://dx.doi.org/10.31234/osf.io/x4azm},
DOI = {10.31234/osf.io/x4azm},
publisher = {Center for Open Science},
author = {Lakens, Daniel and Pahlke, Friedrich and Wassmer, Gernot},
year = {2021},
month = jan
}
@article{scharfstein1997semiparametric,
title={Semiparametric efficiency and its implication on the design and analysis of group-sequential studies},
author={Scharfstein, Daniel O and Tsiatis, Anastasios A and Robins, James M},
journal={Journal of the American Statistical Association},
volume={92},
number={440},
pages={1342--1350},
year={1997},
publisher={Taylor \& Francis Group}
}
@article{jennison1997group,
title={Group-sequential analysis incorporating covariate information},
author={Jennison, Christopher and Turnbull, Bruce W},
journal={Journal of the American Statistical Association},
volume={92},
number={440},
pages={1330--1341},
year={1997},
publisher={Taylor \& Francis}
}
%%% STRATIFIED RANDOMIZATION REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Kahan2011,
doi = {10.1002/sim.4431},
url = {https://doi.org/10.1002/sim.4431},
year = {2011},
month = dec,
publisher = {Wiley},
volume = {31},
number = {4},
pages = {328--340},
author = {Brennan C. Kahan and Tim P. Morris},
title = {Improper analysis of trials randomised using stratified blocks or minimisation},
journal = {Statistics in Medicine}
}
@article{Kernan1999,
doi = {10.1016/s0895-4356(98)00138-3},
url = {https://doi.org/10.1016/s0895-4356(98)00138-3},
year = {1999},
month = jan,
publisher = {Elsevier {BV}},
volume = {52},
number = {1},
pages = {19--26},
author = {W Kernan},
title = {Stratified Randomization for Clinical Trials},
journal = {Journal of Clinical Epidemiology}
}
@article{Lin2015,
doi = {10.1016/j.cct.2015.07.011},
url = {https://doi.org/10.1016/j.cct.2015.07.011},
year = {2015},
month = nov,
publisher = {Elsevier {BV}},
volume = {45},
pages = {21--25},
author = {Yunzhi Lin and Ming Zhu and Zheng Su},
title = {The pursuit of balance: An overview of covariate-adaptive randomization techniques in clinical trials},
journal = {Contemporary Clinical Trials}
}
@article{Wang2021,
doi = {10.1080/01621459.2021.1981338},
url = {https://doi.org/10.1080/01621459.2021.1981338},
year = {2021},
month = nov,
publisher = {Informa {UK} Limited},
volume = {118},
number = {542},
pages = {1152--1163},
author = {Bingkai Wang and Ryoko Susukida and Ramin Mojtabai and Masoumeh Amin-Esmaeili and Michael Rosenblum},
title = {Model-Robust Inference for Clinical Trials that Improve Precision by Stratified Randomization and Covariate Adjustment},
journal = {Journal of the American Statistical Association}
}
%%% SURVEYS OF COVARIATE ADJUSTMENT IN PRACTICE %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Austin2010,
doi = {10.1016/j.jclinepi.2009.06.002},
url = {https://doi.org/10.1016/j.jclinepi.2009.06.002},
year = {2010},
month = feb,
publisher = {Elsevier {BV}},
volume = {63},
number = {2},
pages = {142--153},
author = {Peter C. Austin and Andrea Manca and Merrick Zwarenstein and David N. Juurlink and Matthew B. Stanbrook},
title = {A substantial and confusing variation exists in handling of baseline covariates in randomized controlled trials: a review of trials published in leading medical journals},
journal = {Journal of Clinical Epidemiology}
}
@article{Pocock2002,
doi = {10.1002/sim.1296},
url = {https://doi.org/10.1002/sim.1296},
year = {2002},
publisher = {Wiley},
volume = {21},
number = {19},
pages = {2917--2930},
author = {Stuart J. Pocock and Susan E. Assmann and Laura E. Enos and Linda E. Kasten},
title = {Subgroup analysis, covariate adjustment and baseline comparisons in clinical trial reporting: current practice and problems},
journal = {Statistics in Medicine}
}
%%% REVIEW ARTICLES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Benkeser2020,
doi = {10.1111/biom.13377},
url = {https://doi.org/10.1111/biom.13377},
year = {2020},
month = oct,
publisher = {Wiley},
volume = {77},
number = {4},
pages = {1467--1481},
author = {David Benkeser and Iv{\'{a}}n D{\'{\i}}az and Alex Luedtke and Jodi Segal and Daniel Scharfstein and Michael Rosenblum},
title = {Improving precision and power in randomized trials for {COVID}-19 treatments using covariate adjustment, for binary, ordinal, and time-to-event outcomes},
journal = {Biometrics}
}
%%% COMPARING ESTIMATORS %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Colantuoni2015,
doi = {10.1002/sim.6507},
url = {https://doi.org/10.1002/sim.6507},
year = {2015},
month = apr,
publisher = {Wiley},
volume = {34},
number = {18},
pages = {2602--2617},
author = {Elizabeth Colantuoni and Michael Rosenblum},
title = {Leveraging prognostic baseline variables to gain precision in randomized trials},
journal = {Statistics in Medicine}
}
%%% CONTINUOUS OUTCOMES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Tsiatis2008,
doi = {10.1002/sim.3113},
url = {https://doi.org/10.1002/sim.3113},
year = {2008},
month = oct,
publisher = {Wiley},
volume = {27},
number = {23},
pages = {4658--4677},
author = {Anastasios A. Tsiatis and Marie Davidian and Min Zhang and Xiaomin Lu},
title = {Covariate adjustment for two-sample treatment comparisons in randomized clinical trials: A principled yet flexible approach},
journal = {Statistics in Medicine}
}
@article{Jiang2019,
doi = {10.1080/01621459.2018.1527226},
url = {https://doi.org/10.1080/01621459.2018.1527226},
year = {2019},
month = mar,
publisher = {Informa {UK} Limited},
volume = {114},
number = {528},
pages = {1854--1864},
author = {Fei Jiang and Lu Tian and Haoda Fu and Takahiro Hasegawa and L. J. Wei},
title = {Robust Alternatives to {ANCOVA} for Estimating the Treatment Effect via a Randomized Comparative Study},
journal = {Journal of the American Statistical Association}
}
@article{Zhang2008,
doi = {10.1111/j.1541-0420.2007.00976.x},
url = {https://doi.org/10.1111/j.1541-0420.2007.00976.x},
year = {2008},
month = jan,
publisher = {Wiley},
volume = {64},
number = {3},
pages = {707--715},
author = {Min Zhang and Anastasios A. Tsiatis and Marie Davidian},
title = {Improving Efficiency of Inferences in Randomized Clinical Trials Using Auxiliary Covariates},
journal = {Biometrics}
}
%%% BINARY OUTCOMES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Ge2011,
doi = {10.1177/009286151104500409},
url = {https://doi.org/10.1177/009286151104500409},
year = {2011},
month = jul,
publisher = {Springer Science and Business Media {LLC}},
volume = {45},
number = {4},
pages = {481--493},
author = {Miaomiao Ge and L. Kathryn Durham and R. Daniel Meyer and Wangang Xie and Neal Thomas},
title = {Covariate-Adjusted Difference in Proportions from Clinical Trials Using Logistic Regression and Weighted Risk Differences},
journal = {Drug Information Journal}
}
@article{Moore2009_stat_med,
doi = {10.1002/sim.3445},
url = {https://doi.org/10.1002/sim.3445},
year = {2009},
month = jan,
publisher = {Wiley},
volume = {28},
number = {1},
pages = {39--64},
author = {K. L. Moore and M. J. van der Laan},
title = {Covariate adjustment in randomized trials with binary outcomes: Targeted maximum likelihood estimation},
journal = {Statistics in Medicine}
}
@article{Steingrimsson2017,
doi = {10.1016/j.cct.2016.12.026},
url = {https://doi.org/10.1016/j.cct.2016.12.026},
year = {2017},
month = mar,
publisher = {Elsevier {BV}},
volume = {54},
pages = {18--24},
author = {Jon Arni Steingrimsson and Daniel F. Hanley and Michael Rosenblum},
title = {Improving precision by adjusting for prognostic baseline variables in randomized trials with binary outcomes, without regression model assumptions},
journal = {Contemporary Clinical Trials}
}
%%% ORDINAL OUTCOMES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Diaz2015,
doi = {10.1111/biom.12450},
url = {https://doi.org/10.1111/biom.12450},
year = {2015},
month = nov,
publisher = {Wiley},
volume = {72},
number = {2},
pages = {422--431},
author = {Iv{\'{a}}n D{\'{\i}}az and Elizabeth Colantuoni and Michael Rosenblum},
title = {Enhanced precision in the analysis of randomized trials with ordinal outcomes},
journal = {Biometrics}
}
@article{Fay2018,
doi = {10.1002/sim.7890},
url = {https://doi.org/10.1002/sim.7890},
year = {2018},
month = jul,
publisher = {Wiley},
volume = {37},
number = {27},
pages = {3991--4006},
author = {Michael P. Fay and Yaakov Malinovsky},
title = {Confidence intervals of the Mann-Whitney parameter that are compatible with the Wilcoxon-Mann-Whitney test},
journal = {Statistics in Medicine}
}
@article{Vermeulen2014,
doi = {10.1002/sim.6386},
url = {https://doi.org/10.1002/sim.6386},
year = {2014},
month = dec,
publisher = {Wiley},
volume = {34},
number = {6},
pages = {1012--1030},
author = {Karel Vermeulen and Olivier Thas and Stijn Vansteelandt},
title = {Increasing the power of the Mann-Whitney test in randomized experiments through flexible covariate adjustment},
journal = {Statistics in Medicine}
}
@article{Zhao2008,
doi = {10.1002/sim.2912},
url = {https://doi.org/10.1002/sim.2912},
year = {2008},
publisher = {Wiley},
volume = {27},
number = {3},
pages = {462--468},
author = {Yan D. Zhao and Dewi Rahardja and Yongming Qu},
title = {Sample size calculation for the Wilcoxon{\textendash}Mann{\textendash}Whitney test adjusting for ties},
journal = {Statistics in Medicine}
}
%%% TIME TO EVENT %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Brooks2013,
doi = {10.1515/jci-2013-0001},
url = {https://doi.org/10.1515/jci-2013-0001},
year = {2013},
month = nov,
publisher = {Walter de Gruyter {GmbH}},
volume = {1},
number = {2},
pages = {235--254},
author = {Jordan C. Brooks and Mark J. van der Laan and Daniel E. Singer and Alan S. Go},
title = {Targeted Minimum Loss-Based Estimation of Causal Effects in Right-Censored Survival Data with Time-Dependent Covariates: Warfarin, Stroke, and Death in Atrial Fibrillation},
journal = {Journal of Causal Inference}
}
@article{Lin2017,
doi = {10.1016/j.conctc.2017.09.004},
url = {https://doi.org/10.1016/j.conctc.2017.09.004},
year = {2017},
month = dec,
publisher = {Elsevier {BV}},
volume = {8},
pages = {147--155},
author = {Ray S. Lin and Larry F. Le{\'{o}}n},
title = {Estimation of treatment effects in weighted log-rank tests},
journal = {Contemporary Clinical Trials Communications}
}
@article{Moore2009_j_biopharm_stat,
doi = {10.1080/10543400903243017},
url = {https://doi.org/10.1080/10543400903243017},
year = {2009},
month = oct,
publisher = {Informa {UK} Limited},
volume = {19},
number = {6},
pages = {1099--1131},
author = {K. L. Moore and M. J. van der Laan},
title = {Increasing Power in Randomized Trials with Right Censored Outcomes Through Covariate Adjustment},
journal = {Journal of Biopharmaceutical Statistics}
}
@article{Parast2014,
doi = {10.1080/01621459.2013.842488},
url = {https://doi.org/10.1080/01621459.2013.842488},
year = {2014},
month = jan,
publisher = {Informa {UK} Limited},
volume = {109},
number = {505},
pages = {384--394},
author = {Layla Parast and Lu Tian and Tianxi Cai},
title = {Landmark Estimation of Survival and Treatment Effect in a Randomized Clinical Trial},
journal = {Journal of the American Statistical Association}
}
@article{Stitelman2012,
doi = {10.1515/1557-4679.1334},
url = {https://doi.org/10.1515/1557-4679.1334},
year = {2012},
month = jan,
publisher = {Walter de Gruyter {GmbH}},
volume = {8},
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%%% RESTRICTED MEAN SURVIVAL TIME %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% SANDWICH REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% VIOLATION OF INDEPENDENT INCREMENTS %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% BOOTSTRAP REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% GENERALIZED LINEAR MODELS %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% MISTIE III EXAMPLE %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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%%% CTN-0003 EXAMPLE REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
@article{Ling2009,
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%%% CTN-030 EXAMPLE REFERENCES %%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%%
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