Development and performance of npde for the evaluation of time-to-event models

Autor: Emmanuelle Comets, Marc Lavielle, Marc Cerou, Marylore Chenel, Karl Brendel
Přispěvatelé: Jonchère, Laurent, Centre d'Investigation Clinique [Rennes] (CIC), Université de Rennes (UR)-Hôpital Pontchaillou-Institut National de la Santé et de la Recherche Médicale (INSERM), Infection, Anti-microbiens, Modélisation, Evolution (IAME (UMR_S_1137 / U1137)), Université Paris 13 (UP13)-Université Paris Diderot - Paris 7 (UPD7)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM), Institut de Recherches Internationales Servier [Suresnes] (IRIS), Centre de Mathématiques Appliquées - Ecole Polytechnique (CMAP), École polytechnique (X)-Centre National de la Recherche Scientifique (CNRS), Modélisation en pharmacologie de population (XPOP), École polytechnique (X)-Centre National de la Recherche Scientifique (CNRS)-École polytechnique (X)-Centre National de la Recherche Scientifique (CNRS)-Inria Saclay - Ile de France, Institut National de Recherche en Informatique et en Automatique (Inria)-Institut National de Recherche en Informatique et en Automatique (Inria), Institut de Recherches Internationales Servier., Centre d'Investigation Clinique [Rennes] ( CIC ), Université de Rennes 1 ( UR1 ), Université de Rennes ( UNIV-RENNES ) -Université de Rennes ( UNIV-RENNES ) -Hôpital Pontchaillou-Institut National de la Santé et de la Recherche Médicale ( INSERM ), Infection, Antimicrobiens, Modélisation, Evolution ( IAME ), Université Paris Diderot - Paris 7 ( UPD7 ) -Université Paris 13 ( UP13 ) -Université Sorbonne Paris Cité ( USPC ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ), Institut de Recherches Internationales Servier [Suresnes] ( IRIS ), Centre de Mathématiques Appliquées - Ecole Polytechnique ( CMAP ), École polytechnique ( X ) -Centre National de la Recherche Scientifique ( CNRS ), Modélisation en pharmacologie de population ( XPOP ), Inria Saclay - Ile de France, Institut National de Recherche en Informatique et en Automatique ( Inria ) -Institut National de Recherche en Informatique et en Automatique ( Inria ), Université de Rennes 1 (UR1), Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)-Hôpital Pontchaillou-Institut National de la Santé et de la Recherche Médicale (INSERM), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris 13 (UP13)-Université Paris Diderot - Paris 7 (UPD7)-Université Sorbonne Paris Cité (USPC)
Jazyk: angličtina
Rok vydání: 2018
Předmět:
model evaluation
Time Factors
Datasets as Topic
Pharmaceutical Science
npde
030226 pharmacology & pharmacy
03 medical and health sciences
PSA
0302 clinical medicine
Statistical significance
Statistics
time-to-event
Pharmacology (medical)
[ SDV.BIBS ] Life Sciences [q-bio]/Quantitative Methods [q-bio.QM]
Mathematics
Event (probability theory)
Pharmacology
Clinical Trials as Topic
[SDV.BIBS] Life Sciences [q-bio]/Quantitative Methods [q-bio.QM]
Organic Chemistry
Survival Analysis
Censoring (statistics)
[SDV.BIBS]Life Sciences [q-bio]/Quantitative Methods [q-bio.QM]
Variable (computer science)
Treatment Outcome
Nonlinear Dynamics
Survival function
Sample size determination
Data Interpretation
Statistical
030220 oncology & carcinogenesis
Molecular Medicine
Null hypothesis
Monte Carlo Method
Biomarkers
Software
Biotechnology
Type I and type II errors
Zdroj: Pharmaceutical Research
Pharmaceutical Research, 2018, 35 (2), pp.30. ⟨10.1007/s11095-017-2291-3⟩
Pharmaceutical Research, American Association of Pharmaceutical Scientists, 2018, 35 (2), pp.30. 〈10.1007/s11095-017-2291-3〉
Pharmaceutical Research, American Association of Pharmaceutical Scientists, 2018, 35 (2), pp.30. ⟨10.1007/s11095-017-2291-3⟩
ISSN: 0724-8741
1573-904X
DOI: 10.1007/s11095-017-2291-3⟩
Popis: International audience; Purpose - Normalised prediction distribution errors (npde) are used to graphically and statistically evaluate mixed-effect models for continuous responses. In this study, our aim was to extend npde to time-to-event (TTE) models and evaluate their performance.Methods - Let V denote a dataset with censored TTE observations. The null hypothesis (H) is that observations in V can be described by model M. We extended npde to TTE models using imputations to take into account censoring. We then evaluated their performance in terms of type I error and power to detect model misspecifications for TTE data by means of a simulation study with different sample sizes.Results - Type I error was found to be close to the expected 5% significance level for all sample sizes tested. The npde were able to detect misspecifications in the baseline hazard as well as in the link between the longitudinal variable and the survival function. The ability to detect model misspecifications increased as the difference in the shape of the survival function became more apparent. As expected, the power also increased as the sample size increased. Imputing the censored events tended to decrease the percentage of rejections.Conclusions - We have shown that npde can be readily extended to TTE data and that they perform well with an adequate type I error.
Databáze: OpenAIRE
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