ArticleBMC medical research methodology2024
Determining doses for backfill cohorts based on patient-reported outcome.
Article in BMC medical research methodology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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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
1 citing paper in PubMed.
- BE-BOIN: A Dose Optimization Design Accommodating Backfill and Late-Onset Toxicity.Therapeutic innovation & regulatory science · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
backgroundIncorporating backfill cohorts in phase I oncology trials is a recently developed strategy for dose optimization. However, the efficacy assessment window is long in general, causing a lag in identifying ineffective doses and more patients being backfilled to those doses. There is necessity to investigate how to use patient-reported outcomes (PRO) to determine doses for backfill cohorts.
methodsWe propose a unified Bayesian design framework, called 'Backfill-QoL', to utilize patient-reported quality of life (QoL) data into phase I oncology trials with backfill cohorts, including methods for trial monitoring, algorithm for dose-finding, and criteria for dose selection. Simulation studies and sensitivity analyses are conducted to evaluate the proposed Backfill-QoL design.
resultsThe simulation studies demonstrate that the Backfill-QoL design is more efficient than traditional dose-expansion strategy, and fewer patients would be allocated to doses with unacceptable QoL profiles. A user-friendly Windows desktop application is developed and freely available for implementing the proposed design.
conclusionsThe Backfill-QoL design enables continuous monitoring of safety, efficacy and QoL outcomes, and the recommended phase II dose (RP2D) can be identified in a more patient-centered perspective.
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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.