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Erschienen in: Lifetime Data Analysis 2/2023

07.07.2022

On logistic regression with right censored data, with or without competing risks, and its use for estimating treatment effects

verfasst von: Paul Frédéric Blanche, Anders Holt, Thomas Scheike

Erschienen in: Lifetime Data Analysis | Ausgabe 2/2023

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Abstract

Simple logistic regression can be adapted to deal with right-censoring by inverse probability of censoring weighting (IPCW). We here compare two such IPCW approaches, one based on weighting the outcome, the other based on weighting the estimating equations. We study the large sample properties of the two approaches and show that which of the two weighting methods is the most efficient depends on the censoring distribution. We show by theoretical computations that the methods can be surprisingly different in realistic settings. We further show how to use the two weighting approaches for logistic regression to estimate causal treatment effects, for both observational studies and randomized clinical trials (RCT). Several estimators for observational studies are compared and we present an application to registry data. We also revisit interesting robustness properties of logistic regression in the context of RCTs, with a particular focus on the IPCW weighting. We find that these robustness properties still hold when the censoring weights are correctly specified, but not necessarily otherwise.

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Literatur
Zurück zum Zitat Aalen O, Borgan Ø, Gjessing HK, Gjessing S (2008) Survival and event history analysis: a process point of view. Springer, BerlinCrossRefMATH Aalen O, Borgan Ø, Gjessing HK, Gjessing S (2008) Survival and event history analysis: a process point of view. Springer, BerlinCrossRefMATH
Zurück zum Zitat Andersen PK, Borgan Ø, Gill RD, Keiding N (1993) Statistical Models Based on Counting Processes. Springer, New yorkCrossRefMATH Andersen PK, Borgan Ø, Gill RD, Keiding N (1993) Statistical Models Based on Counting Processes. Springer, New yorkCrossRefMATH
Zurück zum Zitat Azarang L, Scheike T, de Uña-Álvarez J (2017) Direct modeling of regression effects for transition probabilities in the progressive illness-death model. Stat Med 36(12):1964–1976MathSciNet Azarang L, Scheike T, de Uña-Álvarez J (2017) Direct modeling of regression effects for transition probabilities in the progressive illness-death model. Stat Med 36(12):1964–1976MathSciNet
Zurück zum Zitat Bartlett JW (2018) Covariate adjustment and estimation of mean response in randomised trials. Pharmaceutical stat 17(5):648–666 Bartlett JW (2018) Covariate adjustment and estimation of mean response in randomised trials. Pharmaceutical stat 17(5):648–666
Zurück zum Zitat Colantuoni E, Scharfstein DO, Wang C, Hashem MD, Leroux A, Needham DM, Girard TD (2018) Statistical methods to compare functional outcomes in randomized controlled trials with high mortality. BMJ, 360 Colantuoni E, Scharfstein DO, Wang C, Hashem MD, Leroux A, Needham DM, Girard TD (2018) Statistical methods to compare functional outcomes in randomized controlled trials with high mortality. BMJ, 360
Zurück zum Zitat Cortese G, Holmboe SA, Scheike TH (2017) Regression models for the restricted residual mean life for right-censored and left-truncated data. Stat Med 36(11):1803–1822MathSciNetCrossRef Cortese G, Holmboe SA, Scheike TH (2017) Regression models for the restricted residual mean life for right-censored and left-truncated data. Stat Med 36(11):1803–1822MathSciNetCrossRef
Zurück zum Zitat DiRienzo A, Lagakos S (2001) Effects of model misspecification on tests of no randomized treatment effect arising from Cox’s proportional hazards model. J Royal Stat Society: Series B (Statistical Methodology) 63(4):745–757MathSciNetCrossRefMATH DiRienzo A, Lagakos S (2001) Effects of model misspecification on tests of no randomized treatment effect arising from Cox’s proportional hazards model. J Royal Stat Society: Series B (Statistical Methodology) 63(4):745–757MathSciNetCrossRefMATH
Zurück zum Zitat Fine JP, Gray RJ (1999) A proportional hazards model for the subdistribution of a competing risk. J Amer Stat Assoc 94(446):496–509MathSciNetCrossRefMATH Fine JP, Gray RJ (1999) A proportional hazards model for the subdistribution of a competing risk. J Amer Stat Assoc 94(446):496–509MathSciNetCrossRefMATH
Zurück zum Zitat Geskus RB (2016) Data analysis with competing risks and intermediate states. CRC Press Boca Raton Geskus RB (2016) Data analysis with competing risks and intermediate states. CRC Press Boca Raton
Zurück zum Zitat Hernán M, Robins J (2020) Causal Inference: What If. Chapman & Hall/CRC, Boca Raton Hernán M, Robins J (2020) Causal Inference: What If. Chapman & Hall/CRC, Boca Raton
Zurück zum Zitat Holst KK, Scheike T (2021) mets: Analysis of Multivariate Event Times. R package version 1(2):9 Holst KK, Scheike T (2021) mets: Analysis of Multivariate Event Times. R package version 1(2):9
Zurück zum Zitat Holt A, Blanche P, Zareini B, Rajan D, El-Sheikh M, Schjerning A-M, Schou M, Torp-Pedersen C, McGettigan P, Gislason GH et al (2021) Effect of long-term beta-blocker treatment following myocardial infarction among stable, optimally treated patients without heart failure in the reperfusion era: a Danish, nationwide cohort study. Eur Heart J 42(9):907–914CrossRef Holt A, Blanche P, Zareini B, Rajan D, El-Sheikh M, Schjerning A-M, Schou M, Torp-Pedersen C, McGettigan P, Gislason GH et al (2021) Effect of long-term beta-blocker treatment following myocardial infarction among stable, optimally treated patients without heart failure in the reperfusion era: a Danish, nationwide cohort study. Eur Heart J 42(9):907–914CrossRef
Zurück zum Zitat Kim JP (2013) A Note on Using Regression Models to Analyze Randomized Trials: Asymptotically Valid Hypothesis Tests Despite Incorrectly Specified Models. Biometrics 69(1):282–289MathSciNetCrossRefMATH Kim JP (2013) A Note on Using Regression Models to Analyze Randomized Trials: Asymptotically Valid Hypothesis Tests Despite Incorrectly Specified Models. Biometrics 69(1):282–289MathSciNetCrossRefMATH
Zurück zum Zitat Loder E, Groves T, MacAuley D (2010) Registration of observational studies Loder E, Groves T, MacAuley D (2010) Registration of observational studies
Zurück zum Zitat Lok JJ, Yang S, Sharkey B, Hughes MD (2018) Estimation of the cumulative incidence function under multiple dependent and independent censoring mechanisms. Lifetime Data Anal 24(2):201–223MathSciNetCrossRefMATH Lok JJ, Yang S, Sharkey B, Hughes MD (2018) Estimation of the cumulative incidence function under multiple dependent and independent censoring mechanisms. Lifetime Data Anal 24(2):201–223MathSciNetCrossRefMATH
Zurück zum Zitat Luque-Fernandez MA, Schomaker M, Rachet B, Schnitzer ME (2018) Targeted maximum likelihood estimation for a binary treatment: A tutorial. Stat Med 37(16):2530–2546MathSciNetCrossRef Luque-Fernandez MA, Schomaker M, Rachet B, Schnitzer ME (2018) Targeted maximum likelihood estimation for a binary treatment: A tutorial. Stat Med 37(16):2530–2546MathSciNetCrossRef
Zurück zum Zitat Malani HM (1995) A modification of the redistribution to the right algorithm using disease markers. Biometrika 82(3):515–526MathSciNetCrossRefMATH Malani HM (1995) A modification of the redistribution to the right algorithm using disease markers. Biometrika 82(3):515–526MathSciNetCrossRefMATH
Zurück zum Zitat Martens MJ, Logan BR (2020) Group sequential tests for treatment effect on survival and cumulative incidence at a fixed time point. Lifetime Data Anal 26(3):603–623MathSciNetCrossRefMATH Martens MJ, Logan BR (2020) Group sequential tests for treatment effect on survival and cumulative incidence at a fixed time point. Lifetime Data Anal 26(3):603–623MathSciNetCrossRefMATH
Zurück zum Zitat Martinussen T, Vansteelandt S, Andersen PK (2020) Subtleties in the interpretation of hazard contrasts. Lifetime Data Anal 26(4):833–855MathSciNetCrossRefMATH Martinussen T, Vansteelandt S, Andersen PK (2020) Subtleties in the interpretation of hazard contrasts. Lifetime Data Anal 26(4):833–855MathSciNetCrossRefMATH
Zurück zum Zitat McCullagh P, Nelder JA (1989) Generalized Linear Models. Chapman and Hall, London, 2nd edition McCullagh P, Nelder JA (1989) Generalized Linear Models. Chapman and Hall, London, 2nd edition
Zurück zum Zitat Moore KL, van der Laan MJ (2009) Covariate adjustment in randomized trials with binary outcomes: targeted maximum likelihood estimation. Stat Med 28(1):39–64MathSciNetCrossRef Moore KL, van der Laan MJ (2009) Covariate adjustment in randomized trials with binary outcomes: targeted maximum likelihood estimation. Stat Med 28(1):39–64MathSciNetCrossRef
Zurück zum Zitat Ozenne B, Sørensen AL, Scheike T, Torp-Pedersen C, Gerds TA (2017) riskRegression: predicting the risk of an event using Cox regression models. The R J 9(2):440–460CrossRef Ozenne B, Sørensen AL, Scheike T, Torp-Pedersen C, Gerds TA (2017) riskRegression: predicting the risk of an event using Cox regression models. The R J 9(2):440–460CrossRef
Zurück zum Zitat Ozenne BMH, Scheike TH, Stærk L, Gerds TA (2020) On the estimation of average treatment effects with right-censored time to event outcome and competing risks. Biom J 62(3):751–763MathSciNetCrossRefMATH Ozenne BMH, Scheike TH, Stærk L, Gerds TA (2020) On the estimation of average treatment effects with right-censored time to event outcome and competing risks. Biom J 62(3):751–763MathSciNetCrossRefMATH
Zurück zum Zitat Pfeiffer RM, Gail MH (2017) Absolute Risk: Methods and Applications in Clinical Management and Public Health. CRC Press Pfeiffer RM, Gail MH (2017) Absolute Risk: Methods and Applications in Clinical Management and Public Health. CRC Press
Zurück zum Zitat Robins JM, Rotnitzky A (1992) In: Recovery of information and adjustment of dependent censoring using surrogate markers. AIDS Epidemiology-Methodological Issues. Birkhäuser, Boston, pp 24–33 Robins JM, Rotnitzky A (1992) In: Recovery of information and adjustment of dependent censoring using surrogate markers. AIDS Epidemiology-Methodological Issues. Birkhäuser, Boston, pp 24–33
Zurück zum Zitat Robinson LD, Jewell NP (1991) Some surprising results about covariate adjustment in logistic regression models. International Statistical Review/Revue Internationale de Statistique, pp. 227–240 Robinson LD, Jewell NP (1991) Some surprising results about covariate adjustment in logistic regression models. International Statistical Review/Revue Internationale de Statistique, pp. 227–240
Zurück zum Zitat Rosenblum M, Steingrimsson JA (2016) Matching the Efficiency Gains of the Logistic Regression Estimator While Avoiding its Interpretability Problems, in Randomized Trials. Technical report Rosenblum M, Steingrimsson JA (2016) Matching the Efficiency Gains of the Logistic Regression Estimator While Avoiding its Interpretability Problems, in Randomized Trials. Technical report
Zurück zum Zitat Rossello X, Pocock SJ, Julian DG (2015) Long-term use of cardiovascular drugs: challenges for research and for patient care. J Amer Coll Cardiology 66(11):1273–1285CrossRef Rossello X, Pocock SJ, Julian DG (2015) Long-term use of cardiovascular drugs: challenges for research and for patient care. J Amer Coll Cardiology 66(11):1273–1285CrossRef
Zurück zum Zitat Rotnitzky A, Farall A, Bergesio A, Scharfstein D (2007) Analysis of failure time data under competing censoring mechanisms. J Royal Stat Soc: Series B (Stat Methodol) 69(3):307–327MathSciNetCrossRefMATH Rotnitzky A, Farall A, Bergesio A, Scharfstein D (2007) Analysis of failure time data under competing censoring mechanisms. J Royal Stat Soc: Series B (Stat Methodol) 69(3):307–327MathSciNetCrossRefMATH
Zurück zum Zitat Rufibach K (2019) Treatment effect quantification for time-to-event endpoints-Estimands, analysis strategies, and beyond. Pharmaceutical stat 18(2):145–165CrossRef Rufibach K (2019) Treatment effect quantification for time-to-event endpoints-Estimands, analysis strategies, and beyond. Pharmaceutical stat 18(2):145–165CrossRef
Zurück zum Zitat Scharfstein DO, Rotnitzky A, Robins JM (1999) Adjusting for nonignorable drop-out using semiparametric nonresponse models. Journal of the American Statistical Association, 94(448):1096–1120 (with Rejoinder, 1135–1146) Scharfstein DO, Rotnitzky A, Robins JM (1999) Adjusting for nonignorable drop-out using semiparametric nonresponse models. Journal of the American Statistical Association, 94(448):1096–1120 (with Rejoinder, 1135–1146)
Zurück zum Zitat Scheike T, Zhang M, Gerds T (2008) Predicting cumulative incidence probability by direct binomial regression. Biometrika 95:205–220MathSciNetCrossRefMATH Scheike T, Zhang M, Gerds T (2008) Predicting cumulative incidence probability by direct binomial regression. Biometrika 95:205–220MathSciNetCrossRefMATH
Zurück zum Zitat Schumacher M, Ohneberg K, Beyersmann J (2016) Competing risk bias was common in a prominent medical journal. Journal of Clinical Epidemiology 80:135–136CrossRef Schumacher M, Ohneberg K, Beyersmann J (2016) Competing risk bias was common in a prominent medical journal. Journal of Clinical Epidemiology 80:135–136CrossRef
Zurück zum Zitat Stensrud MJ, Hernán MA (2020) Why test for proportional hazards? Jama 323(14):1401–1402CrossRef Stensrud MJ, Hernán MA (2020) Why test for proportional hazards? Jama 323(14):1401–1402CrossRef
Zurück zum Zitat Sutradhar R, Austin PC (2018) Relative rates not relative risks: addressing a widespread misinterpretation of hazard ratios. Ann epidemiology 28(1):54–57CrossRef Sutradhar R, Austin PC (2018) Relative rates not relative risks: addressing a widespread misinterpretation of hazard ratios. Ann epidemiology 28(1):54–57CrossRef
Zurück zum Zitat Tsiatis A (2006) Semiparametric theory and missing data. Springer Science & Business Media Tsiatis A (2006) Semiparametric theory and missing data. Springer Science & Business Media
Zurück zum Zitat Uno H, Cai T, Tian L, Wei L (2007) Evaluating prediction rules for t-year survivors with censored regression models. J Amer Stat Assoc 102(478):527–537MathSciNetCrossRefMATH Uno H, Cai T, Tian L, Wei L (2007) Evaluating prediction rules for t-year survivors with censored regression models. J Amer Stat Assoc 102(478):527–537MathSciNetCrossRefMATH
Zurück zum Zitat Van der Laan MJ, Rose S (2011) Targeted learning: causal inference for observational and experimental data. Springer Science & Business Media Van der Laan MJ, Rose S (2011) Targeted learning: causal inference for observational and experimental data. Springer Science & Business Media
Zurück zum Zitat Van Der Laan MJ, Rubin D (2006) Targeted maximum likelihood learning. The international journal of biostatistics, 2(1) Van Der Laan MJ, Rubin D (2006) Targeted maximum likelihood learning. The international journal of biostatistics, 2(1)
Zurück zum Zitat Vansteelandt S, Martinussen T, Tchetgen ET (2014) On adjustment for auxiliary covariates in additive hazard models for the analysis of randomized experiments. Biometrika 101(1):237–244MathSciNetCrossRefMATH Vansteelandt S, Martinussen T, Tchetgen ET (2014) On adjustment for auxiliary covariates in additive hazard models for the analysis of randomized experiments. Biometrika 101(1):237–244MathSciNetCrossRefMATH
Zurück zum Zitat Wang B, Susukida R, Mojtabai R, Amin-Esmaeili M, Rosenblum M (2021) Model-robust inference for clinical trials that improve precision by stratified randomization and covariate adjustment. Journal of the American Statistical Association, pp. 1–12 Wang B, Susukida R, Mojtabai R, Amin-Esmaeili M, Rosenblum M (2021) Model-robust inference for clinical trials that improve precision by stratified randomization and covariate adjustment. Journal of the American Statistical Association, pp. 1–12
Zurück zum Zitat Young JG, Stensrud MJ, Tchetgen Tchetgen EJ, Hernán MA (2020) A causal framework for classical statistical estimands in failure-time settings with competing events. Stat Med 39(8):1199–1236MathSciNetCrossRef Young JG, Stensrud MJ, Tchetgen Tchetgen EJ, Hernán MA (2020) A causal framework for classical statistical estimands in failure-time settings with competing events. Stat Med 39(8):1199–1236MathSciNetCrossRef
Zurück zum Zitat Zhang M, Tsiatis AA, Davidian M (2008) Improving efficiency of inferences in randomized clinical trials using auxiliary covariates. Biometrics 64(3):707–715MathSciNetCrossRefMATH Zhang M, Tsiatis AA, Davidian M (2008) Improving efficiency of inferences in randomized clinical trials using auxiliary covariates. Biometrics 64(3):707–715MathSciNetCrossRefMATH
Zurück zum Zitat Zhang X, Zhang M-J (2011) SAS macros for estimation of direct adjusted cumulative incidence curves under proportional subdistribution hazards models. Comput methods programs in biomedicine 101(1):87–93CrossRef Zhang X, Zhang M-J (2011) SAS macros for estimation of direct adjusted cumulative incidence curves under proportional subdistribution hazards models. Comput methods programs in biomedicine 101(1):87–93CrossRef
Zurück zum Zitat Zheng Y, Cai T, Feng Z (2006) Application of the time-dependent ROC curves for prognostic accuracy with multiple biomarkers. Biometrics 62(1):279–287MathSciNetCrossRefMATH Zheng Y, Cai T, Feng Z (2006) Application of the time-dependent ROC curves for prognostic accuracy with multiple biomarkers. Biometrics 62(1):279–287MathSciNetCrossRefMATH
Metadaten
Titel
On logistic regression with right censored data, with or without competing risks, and its use for estimating treatment effects
verfasst von
Paul Frédéric Blanche
Anders Holt
Thomas Scheike
Publikationsdatum
07.07.2022
Verlag
Springer US
Erschienen in
Lifetime Data Analysis / Ausgabe 2/2023
Print ISSN: 1380-7870
Elektronische ISSN: 1572-9249
DOI
https://doi.org/10.1007/s10985-022-09564-6

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