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A benchmark for dose-finding studies with unknown ordering
Mozgunov, Pavel, Paoletti, Xavier und Jaki, Thomas
(2021)
A benchmark for dose-finding studies with unknown ordering.
Biostatistics 23 (3), S. 721-737.
Veröffentlichungsdatum dieses Volltextes: 30 Sep 2025 06:01
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.77859
Zusammenfassung
An important tool to evaluate the performance of a dose-finding design is the nonparametric optimal benchmark that provides an upper bound on the performance of a design under a given scenario. A fundamental assumption of the benchmark is that the investigator can arrange doses in a monotonically increasing toxicity order. While the benchmark can be still applied to combination studies in which ...
An important tool to evaluate the performance of a dose-finding design is the nonparametric optimal benchmark that provides an upper bound on the performance of a design under a given scenario. A fundamental assumption of the benchmark is that the investigator can arrange doses in a monotonically increasing toxicity order. While the benchmark can be still applied to combination studies in which not all dose combinations can be ordered, it does not account for the uncertainty in the ordering. In this article, we propose a generalization of the benchmark that accounts for this uncertainty and, as a result, provides a sharper upper bound on the performance. The benchmark assesses how probable the occurrence of each ordering is, given the complete information about each patient. The proposed approach can be applied to trials with an arbitrary number of endpoints with discrete or continuous distributions. We illustrate the utility of the benchmark using recently proposed dose-finding designs for Phase I combination trials with a binary toxicity endpoint and Phase I/II combination trials with binary toxicity and continuous efficacy.
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Details
| Dokumentenart | Artikel | ||||
| Titel eines Journals oder einer Zeitschrift | Biostatistics | ||||
| Verlag: | Oxford Academic, Oxford University Press | ||||
|---|---|---|---|---|---|
| Band: | 23 | ||||
| Nummer des Zeitschriftenheftes oder des Kapitels: | 3 | ||||
| Seitenbereich: | S. 721-737 | ||||
| Datum | 4 Januar 2021 | ||||
| Institutionen | Informatik und Data Science > Fachbereich Maschinelles Lernen und Data Science > Chair for Computational Statistics (Prof. Dr. Thomas Jaki) | ||||
| Identifikationsnummer |
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| Stichwörter / Keywords | Benchmark; Combination trial; Dose finding; Partial ordering; Power likelihood | ||||
| Dewey-Dezimal-Klassifikation | 000 Informatik, Informationswissenschaft, allgemeine Werke > 004 Informatik | ||||
| Status | Veröffentlicht | ||||
| Begutachtet | Ja, diese Version wurde begutachtet | ||||
| An der Universität Regensburg entstanden | Nein | ||||
| URN der UB Regensburg | urn:nbn:de:bvb:355-epub-778596 | ||||
| Dokumenten-ID | 77859 |
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