ArticleStatistics in medicine2020
Incorporating patient-reported outcomes in dose-finding clinical trials.
Article in Statistics in medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed.
- Measuring toxicity better: patient-reported outcome-informed calibration for CTCAE assessment.Translational cancer research · 2026Article
- Illustrative Integration of Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) Data in Dose-Cohort Review for a Phase I Clinical Trial.Research square · 2026Article
- Generalized Pairwise Comparisons in Dose Optimization Oncology Trials: Beyond Safety to Multi-outcome Dose Selection.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
- PRO-ADD: Patient-empowered dose-finding trials integrating safety, preliminary efficacy and patient-reported outcomes for optimal dose selection.Statistical methods in medical research · 2026Article
- International Consensus-Driven Recommendations for Patient-Reported Outcome Research Objectives in Early Phase Dose-Finding Oncology Trials: OPTIMISE-ROR.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2026Article
- Understanding the Role of Patient-Reported Outcomes for Decision-Making in Early-Phase Dose-Finding Clinical Trials.Current oncology (Toronto, Ont.) · 2025Article
- Isotonic Phase I cancer clinical trial design utilizing patient-reported outcomes.Statistics in biopharmaceutical research · 2025Article
- Determining doses for backfill cohorts based on patient-reported outcome.BMC medical research methodology · 2024Article
- Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable.EClinicalMedicine · 2024Review
- Patient and public involvement and engagement in the development of innovative patient-centric early phase dose-finding trial designs.Research involvement and engagement · 2024Article
- Realizing the promise of Project Optimus: Challenges and emerging opportunities for dose optimization in oncology drug development.CPT: pharmacometrics & systems pharmacology · 2024Review
- International Society for Quality of Life Research commentary on the US Food and Drug Administration draft guidance for industry on core patient-reported outcomes in cancer clinical trials.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2023Article
- Application of the patient-reported outcomes continual reassessment method to a phase I study of radiotherapy in endometrial cancer.The international journal of biostatistics · 2023Article
- Designing Dose-Finding Phase I Clinical Trials: Top 10 Questions That Should Be Discussed With Your Statistician.JCO precision oncology · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Oncology dose-finding clinical trials determine the maximum tolerated dose (MTD) based on toxicity outcomes captured by clinicians. With the availability of more rigorous instruments for measuring toxicity directly from patients, there is a growing interest to incorporate patient-reported outcomes (PRO) in clinical trials to inform patient tolerability. This is particularly important for dose-finding trials to ensure the identification of a well-tolerated dose. In this paper, we propose three extensions of the continual reassessment method (CRM), termed PRO-CRMs, that incorporate both clinician and patient outcomes. The first method is a marginal modeling approach whereby clinician and patient toxicity outcomes are modeled separately. The other two methods impose a constraint using a joint outcome defined based on both clinician and patient toxicities and model them either jointly or marginally. Simulation studies show that while all three PRO-CRMs select well-tolerated doses based on clinician's and patient's perspectives, the methods using a joint outcome perform better and have similar performance. We also show that the proposed PRO-CRMs are consistent under robust model assumptions.
Indexed as
Identifiers
What OpenQuestion holds
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.