Methods for non-proportional hazards in clinical trials: A systematic review

Fuente: arXiv
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Autori principali: Bardo, Maximilian, Huber, Cynthia, Benda, Norbert, Brugger, Jonas, Fellinger, Tobias, Galaune, Vaidotas, Heinz, Judith, Heinzl, Harald, Hooker, Andrew C., Klinglmüller, Florian, König, Franz, Mathes, Tim, Mittlböck, Martina, Posch, Martin, Ristl, Robin, Friede, Tim
Natura: Preprint
Pubblicazione: 2023
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author Bardo, Maximilian
Huber, Cynthia
Benda, Norbert
Brugger, Jonas
Fellinger, Tobias
Galaune, Vaidotas
Heinz, Judith
Heinzl, Harald
Hooker, Andrew C.
Klinglmüller, Florian
König, Franz
Mathes, Tim
Mittlböck, Martina
Posch, Martin
Ristl, Robin
Friede, Tim
author_facet Bardo, Maximilian
Huber, Cynthia
Benda, Norbert
Brugger, Jonas
Fellinger, Tobias
Galaune, Vaidotas
Heinz, Judith
Heinzl, Harald
Hooker, Andrew C.
Klinglmüller, Florian
König, Franz
Mathes, Tim
Mittlböck, Martina
Posch, Martin
Ristl, Robin
Friede, Tim
contents For the analysis of time-to-event data, frequently used methods such as the log-rank test or the Cox proportional hazards model are based on the proportional hazards assumption, which is often debatable. Although a wide range of parametric and non-parametric methods for non-proportional hazards (NPH) has been proposed, there is no consensus on the best approaches. To close this gap, we conducted a systematic literature search to identify statistical methods and software appropriate under NPH. Our literature search identified 907 abstracts, out of which we included 211 articles, mostly methodological ones. Review articles and applications were less frequently identified. The articles discuss effect measures, effect estimation and regression approaches, hypothesis tests, and sample size calculation approaches, which are often tailored to specific NPH situations. Using a unified notation, we provide an overview of methods available. Furthermore, we derive some guidance from the identified articles. We summarized the contents from the literature review in a concise way in the main text and provide more detailed explanations in the supplement.
format Preprint
id arxiv_https___arxiv_org_abs_2306_16858
institution arXiv
publishDate 2023
record_format arxiv
spellingShingle Methods for non-proportional hazards in clinical trials: A systematic review
Bardo, Maximilian
Huber, Cynthia
Benda, Norbert
Brugger, Jonas
Fellinger, Tobias
Galaune, Vaidotas
Heinz, Judith
Heinzl, Harald
Hooker, Andrew C.
Klinglmüller, Florian
König, Franz
Mathes, Tim
Mittlböck, Martina
Posch, Martin
Ristl, Robin
Friede, Tim
Methodology
Applications
62Nxx, 62Pxx
For the analysis of time-to-event data, frequently used methods such as the log-rank test or the Cox proportional hazards model are based on the proportional hazards assumption, which is often debatable. Although a wide range of parametric and non-parametric methods for non-proportional hazards (NPH) has been proposed, there is no consensus on the best approaches. To close this gap, we conducted a systematic literature search to identify statistical methods and software appropriate under NPH. Our literature search identified 907 abstracts, out of which we included 211 articles, mostly methodological ones. Review articles and applications were less frequently identified. The articles discuss effect measures, effect estimation and regression approaches, hypothesis tests, and sample size calculation approaches, which are often tailored to specific NPH situations. Using a unified notation, we provide an overview of methods available. Furthermore, we derive some guidance from the identified articles. We summarized the contents from the literature review in a concise way in the main text and provide more detailed explanations in the supplement.
title Methods for non-proportional hazards in clinical trials: A systematic review
topic Methodology
Applications
62Nxx, 62Pxx
url https://arxiv.org/abs/2306.16858