Evaluation of Treatment Effect with Paired Failure Times in a Single-Arm Phase II Trial in Oncology
Joint Authors
Texier, Matthieu
Rotolo, Federico
Ducreux, Michel
Bouché, Olivier
Pignon, Jean-Pierre
Michiels, Stefan
Source
Computational and Mathematical Methods in Medicine
Issue
Vol. 2018, Issue 2018 (31 Dec. 2018), pp.1-8, 8 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2018-01-11
Country of Publication
Egypt
No. of Pages
8
Main Subjects
Abstract EN
In early phase clinical trials of cytotoxic drugs in oncology, the efficacy is typically evaluated based on the tumor shrinkage.
However, this criterion is not always appropriate for more recent cytostatic agents, and alternative endpoints have been proposed.
The growth modulation index (GMI), defined as the ratio between the times to progression in two successive treatment lines, has been proposed for a single-arm phase II trials.
The treatment effect is evaluated by estimating the rate of patients having a GMI superior to a given threshold.
To estimate this rate, we investigated a parametric method based on the distribution of the times to progression and a nonparametric one based on a midrank estimator.
Through simulations, we studied their operating characteristics and the impact of different design parameters (censoring, dependence, and distribution) on them.
In these simulations, the nonparametric estimator slightly underestimated the rate and had slightly overconservative confidence intervals in some cases.
Conversely, the parametric estimator overestimated the rate and had anticonservative confidence intervals in some cases.
The nonparametric method appeared to be more robust to censoring than the parametric one.
In conclusion, we recommend the nonparametric method, but the parametric method can be used as a supplementary tool.
American Psychological Association (APA)
Texier, Matthieu& Rotolo, Federico& Ducreux, Michel& Bouché, Olivier& Pignon, Jean-Pierre& Michiels, Stefan. 2018. Evaluation of Treatment Effect with Paired Failure Times in a Single-Arm Phase II Trial in Oncology. Computational and Mathematical Methods in Medicine،Vol. 2018, no. 2018, pp.1-8.
https://search.emarefa.net/detail/BIM-1131797
Modern Language Association (MLA)
Texier, Matthieu…[et al.]. Evaluation of Treatment Effect with Paired Failure Times in a Single-Arm Phase II Trial in Oncology. Computational and Mathematical Methods in Medicine No. 2018 (2018), pp.1-8.
https://search.emarefa.net/detail/BIM-1131797
American Medical Association (AMA)
Texier, Matthieu& Rotolo, Federico& Ducreux, Michel& Bouché, Olivier& Pignon, Jean-Pierre& Michiels, Stefan. Evaluation of Treatment Effect with Paired Failure Times in a Single-Arm Phase II Trial in Oncology. Computational and Mathematical Methods in Medicine. 2018. Vol. 2018, no. 2018, pp.1-8.
https://search.emarefa.net/detail/BIM-1131797
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1131797