ArticleStatistics in medicine2021
Precision Bayesian phase I-II dose-finding based on utilities tailored to prognostic subgroups.
Article in Statistics in medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 21 citations in OpenAlex.
- A Bayesian phase I/II design to determine subgroup-specific optimal dose for immunotherapy sequentially combined with radiotherapy.Pharmaceutical statistics · 2023Trial
- BAR12: Bayesian Autoregressive Phase 1-2 Design for Cell Therapy Trials With Manufacturing Changes.Statistics in medicine · 2026Article
- Precision generalized phase I-II designs.Biometrics · 2025Article
- Bayesian Safety and Futility Monitoring in Phase II Trials Using One Utility-Based Rule.Statistics in medicine · 2024Article
- Risk-benefit trade-offs and precision utilities in phase I-II clinical trials.Clinical trials (London, England) · 2024Article
- Adaptive Bayesian information borrowing methods for finding and optimizing subgroup-specific doses.Clinical trials (London, England) · 2024Review
- Adaptive phase I-II clinical trial designs identifying optimal biological doses for targeted agents and immunotherapies.Clinical trials (London, England) · 2024Review
- Novel Clinical Trial Designs with Dose Optimization to Improve Long-term Outcomes.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023Review
- How large must a dose-optimization trial be?CPT: pharmacometrics & systems pharmacology · 2023Article
- Interpreting Randomized Controlled Trials.Cancers · 2023Review
- Bayesian treatment screening and selection using subgroup-specific utilities of response and toxicity.Biometrics · 2023Article
- Less is More? First Impressions From COSMIC-313.Cancer investigation · 2023Article
- Review
- BIPSE: A biomarker-based phase I/II design for immunotherapy trials with progression-free survival endpoint.Statistics in medicine · 2022Article
- Validation of Prognostic Scores in Patients With Metastatic Urothelial Cancer Enrolling in Phase I Targeted Therapy or Next Generation Immunotherapy Trials.Clinical genitourinary cancer · 2022Article
- A Personalized Dose-Finding Algorithm Based on Adaptive Gaussian Process Regression.Pharmaceutical statisticsArticle
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
A Bayesian phase I-II design is presented that optimizes the dose of a new agent within predefined prognostic subgroups. The design is motivated by a trial to evaluate targeted agents for treating metastatic clear cell renal carcinoma, where a prognostic risk score defined by clinical variables and biomarkers is well established. Two clinical outcomes are used for dose-finding, time-to-toxicity during a prespecified follow-up period, and efficacy characterized by ordinal disease status evaluated at the end of follow-up. A joint probability model is constructed for these outcomes as functions of dose and subgroup. The model performs adaptive clustering of adjacent subgroups having similar dose-outcome distributions to facilitate borrowing information across subgroups. To quantify toxicity-efficacy risk-benefit trade-offs that may differ between subgroups, the objective function is based on outcome utilities elicited separately for each subgroup. In the context of the renal cancer trial, a design is constructed and a simulation study is presented to evaluate the design's reliability, safety, and robustness, and to compare it to designs that either ignore subgroups or run a separate trial within each subgroup.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.