Analyzing differences between restricted mean survival time curves using pseudo-values
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Analyzing differences between restricted mean survival time curves using pseudo-values. / Ambrogi, Federico; Iacobelli, Simona; Andersen, Per Kragh.
In: BMC Medical Research Methodology, Vol. 22, No. 1, 71, 2022.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Analyzing differences between restricted mean survival time curves using pseudo-values
AU - Ambrogi, Federico
AU - Iacobelli, Simona
AU - Andersen, Per Kragh
PY - 2022
Y1 - 2022
N2 - Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully elucidate study results. Restricted mean survival time (RMST) differences between groups have been advocated as useful measures of association. Recent work focused on model-free estimates of the difference in restricted mean survival through follow-up times, instead of focusing on a single time horizon. The resulting curve can be used to quantify the association in time units with a simultaneous confidence band. In this work a model-based estimate of the curve is proposed using pseudo-values allowing for possible covariate adjustment. The method is easily implementable with available software and makes possible to compute a simultaneous confidence region for the curve. The pseudo-values regression using multiple restriction times is in good agreement with the estimates obtained by standard direct regression models fixing a single restriction time. Moreover, the proposed method is flexible enough to reproduce the results of the non-parametric approach when no covariates are considered. Examples where it is important to adjust for baseline covariates will be used to illustrate the different methods together with some simulations.
AB - Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully elucidate study results. Restricted mean survival time (RMST) differences between groups have been advocated as useful measures of association. Recent work focused on model-free estimates of the difference in restricted mean survival through follow-up times, instead of focusing on a single time horizon. The resulting curve can be used to quantify the association in time units with a simultaneous confidence band. In this work a model-based estimate of the curve is proposed using pseudo-values allowing for possible covariate adjustment. The method is easily implementable with available software and makes possible to compute a simultaneous confidence region for the curve. The pseudo-values regression using multiple restriction times is in good agreement with the estimates obtained by standard direct regression models fixing a single restriction time. Moreover, the proposed method is flexible enough to reproduce the results of the non-parametric approach when no covariates are considered. Examples where it is important to adjust for baseline covariates will be used to illustrate the different methods together with some simulations.
KW - RMST curve difference
KW - Pseudo-values
KW - Crossing survival curves
KW - ADJUVANT THERAPY
KW - HAZARD RATIO
KW - LIFE
KW - FLUOROURACIL
KW - LEVAMISOLE
KW - MODELS
U2 - 10.1186/s12874-022-01559-z
DO - 10.1186/s12874-022-01559-z
M3 - Journal article
C2 - 35300614
VL - 22
JO - B M C Medical Research Methodology
JF - B M C Medical Research Methodology
SN - 1471-2288
IS - 1
M1 - 71
ER -
ID: 302379899