guest ::
| Home > Publications database > The Progression-Free-Survival Ratio in Molecularly Aided Tumor Trials: A Critical Examination of Current Practice and Suggestions for Alternative Methods. |
| Home > Publications database > The Progression-Free-Survival Ratio in Molecularly Aided Tumor Trials: A Critical Examination of Current Practice and Suggestions for Alternative Methods. |
| Journal Article | DKFZ-2024-02737 |
; ; ; ;
2025
Wiley-VCH
Berlin
This record in other databases: 
Please use a persistent id in citations: doi:10.1002/bimj.70028
Abstract: The progression-free-survival ratio is a popular endpoint in oncology trials, which is frequently applied to evaluate the efficacy of molecularly targeted treatments in late-stage patients. Using elementary calculations and simulations, numerous shortcomings of the current methodology are pointed out. As a remedy to these shortcomings, an alternative methodology is proposed, using a marginal Cox model or a marginal accelerated failure time model for clustered time-to-event data. Using comprehensive simulations, it is shown that this methodology outperforms existing methods in settings where the intrapatient correlation is low to moderate. The performance of the model is further demonstrated in a real data example from a molecularly aided tumor trial. Sample size considerations are discussed.
Keyword(s): Humans (MeSH) ; Biometry: methods (MeSH) ; Neoplasms: mortality (MeSH) ; Clinical Trials as Topic: methods (MeSH) ; Progression-Free Survival (MeSH) ; Molecular Targeted Therapy: methods (MeSH) ; Models, Statistical (MeSH) ; Proportional Hazards Models (MeSH) ; Weibull distribution ; accelerated failure time model ; growth modulation index ; marginal model ; paired time‐to‐event data ; progression‐free‐survival ratio
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; DEAL Wiley ; Essential Science Indicators ; IF < 5 ; JCR ; Nationallizenz
; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection
|
The record appears in these collections: |
