Dose finding studies for therapies with late-onset toxicities: A comparison study of designs.


Journal

Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016

Informations de publication

Date de publication:
30 12 2022
Historique:
revised: 28 07 2022
received: 30 04 2021
accepted: 03 10 2022
pubmed: 18 10 2022
medline: 16 12 2022
entrez: 17 10 2022
Statut: ppublish

Résumé

An objective of phase I dose-finding trials is to find the maximum tolerated dose; the dose with a particular risk of toxicity. Frequently, this risk is assessed across the first cycle of therapy. However, in oncology, a course of treatment frequently consists of multiple cycles of therapy. In many cases, the overall risk of toxicity for a given treatment is not fully encapsulated by observations from the first cycle, and hence it is advantageous to include toxicity outcomes from later cycles in phase I trials. Extending the follow up period in a trial naturally extends the total length of the trial which is undesirable. We present a comparison of eight methods that incorporate late onset toxicities while not extensively extending the trial length. We conduct simulation studies over a number of scenarios and in two settings; the first setting with minimal stopping rules and the second setting with a full set of standard stopping rules expected in such a dose finding study. We find that the model-based approaches in general outperform the model-assisted approaches, with an interval censored approach and a modified version of the time-to-event continual reassessment method giving the most promising overall performance in terms of correct selections and trial length. Further recommendations are made for the implementation of such methods.

Identifiants

pubmed: 36250912
doi: 10.1002/sim.9593
pmc: PMC10092569
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

5767-5788

Subventions

Organisme : Department of Health
ID : NIHR-SRF-2015-08-001
Pays : United Kingdom
Organisme : Department of Health
ID : BRC-1215-20014
Pays : United Kingdom
Organisme : Medical Research Council
ID : MC_UU_00002/14
Pays : United Kingdom

Informations de copyright

© 2022 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd.

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Auteurs

Helen Barnett (H)

MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Learning Development, Lancaster University, Lancaster, UK.

Oliver Boix (O)

Bayer AG, Leverkusen, Germany.

Dimitris Kontos (D)

ClinBAY, Limassol, Cyprus.

Thomas Jaki (T)

MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Faculty of Informatics and Data Science, University of Regensburg, Regensburg, Germany.

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