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Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer
This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determine the maximum tolerated dose (MTD) at the study conclusion. We describe an application of a Bayesian fo...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
De Gruyter
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238853/ https://www.ncbi.nlm.nih.gov/pubmed/36394530 http://dx.doi.org/10.1515/ijb-2022-0023 |
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author | Wages, Nolan A. Nelson, Bailey Kharofa, Jordan Meier, Teresa |
author_facet | Wages, Nolan A. Nelson, Bailey Kharofa, Jordan Meier, Teresa |
author_sort | Wages, Nolan A. |
collection | PubMed |
description | This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determine the maximum tolerated dose (MTD) at the study conclusion. We describe an application of a Bayesian form of the patient-reported outcomes continual reassessment method (PRO-CRMB) in an ongoing Phase I study of adjuvant hypofractionated whole pelvis radiation therapy (WPRT) in endometrial cancer (NCT04458402). The study’s primary objective is to determine the MTD per fraction of WPRT, defined by acceptable clinician- and patient-reported DLT rates. We conduct simulation studies of the operating characteristics of the design and compared them to a rule-based approach. We illustrate that the PRO-CRMB makes appropriate dose assignments during the study to give investigators and reviewers an idea of how the method behaves. In simulation studies, the PRO-CRMB demonstrates superior performance to a 5 + 2 stepwise design in terms of recommending target treatment courses and allocating patients to these courses. The design is accompanied by an easy-to-use R shiny web application to simulate operating characteristics at the design stage and sequentially update dose assignments throughout the trial’s conduct. |
format | Online Article Text |
id | pubmed-10238853 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | De Gruyter |
record_format | MEDLINE/PubMed |
spelling | pubmed-102388532023-06-04 Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer Wages, Nolan A. Nelson, Bailey Kharofa, Jordan Meier, Teresa Int J Biostat Article This article considers the concept of designing Phase I clinical trials using both clinician- and patient-reported outcomes to adaptively allocate study participants to tolerable doses and determine the maximum tolerated dose (MTD) at the study conclusion. We describe an application of a Bayesian form of the patient-reported outcomes continual reassessment method (PRO-CRMB) in an ongoing Phase I study of adjuvant hypofractionated whole pelvis radiation therapy (WPRT) in endometrial cancer (NCT04458402). The study’s primary objective is to determine the MTD per fraction of WPRT, defined by acceptable clinician- and patient-reported DLT rates. We conduct simulation studies of the operating characteristics of the design and compared them to a rule-based approach. We illustrate that the PRO-CRMB makes appropriate dose assignments during the study to give investigators and reviewers an idea of how the method behaves. In simulation studies, the PRO-CRMB demonstrates superior performance to a 5 + 2 stepwise design in terms of recommending target treatment courses and allocating patients to these courses. The design is accompanied by an easy-to-use R shiny web application to simulate operating characteristics at the design stage and sequentially update dose assignments throughout the trial’s conduct. De Gruyter 2022-11-17 /pmc/articles/PMC10238853/ /pubmed/36394530 http://dx.doi.org/10.1515/ijb-2022-0023 Text en © 2022 the author(s), published by De Gruyter, Berlin/Boston https://creativecommons.org/licenses/by/4.0/This work is licensed under the Creative Commons Attribution 4.0 International License. |
spellingShingle | Article Wages, Nolan A. Nelson, Bailey Kharofa, Jordan Meier, Teresa Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title | Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title_full | Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title_fullStr | Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title_full_unstemmed | Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title_short | Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer |
title_sort | application of the patient-reported outcomes continual reassessment method to a phase i study of radiotherapy in endometrial cancer |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238853/ https://www.ncbi.nlm.nih.gov/pubmed/36394530 http://dx.doi.org/10.1515/ijb-2022-0023 |
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