Evidence map›Paper›PMID 39030498›Full record

ArticleBMC medical research methodology2024

Bayesian sequential monitoring strategies for trials of digestive cancer therapeutics.

Guillaume Mulier, Ruitao Lin, Thomas Aparicio, Lucie Biard

Abstract read
In one paragraph

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.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Guillaume MulierECSTRRA team UMR 1153, INSERM, Saint-Louis hospital, 1 avenue Claude Vellefaux, Paris, 75010, France. guillaume.mulier@u-paris.fr.
Ruitao LinDepartment of Biostatistics, MD Anderson Cancer Center, 7007 Bertner Avenue, Houston, 77030, Texas, USA.
Thomas AparicioService d'hépato-gastro-entérologie, Hôpital Saint-Louis, 1 avenue Claude Vellefaux, Paris, 75010, France.
Lucie BiardECSTRRA team UMR 1153, INSERM, Saint-Louis hospital, 1 avenue Claude Vellefaux, Paris, 75010, France.

Funding

Bayesian Methods for Complex Precision Biotherapy Trials in OncologyR01CA261978 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI LIN, RUITAO, THALL, PETER F · 2021 to 2024
$1.4M
Novel designs for multi-arm multi-dose multi-stage platform trialsR21LM014699 · NLM · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Ruitao Lin · 2025 to 2026
$405k
NCI NIH HHS R01 CA261978NLM NIH HHS R21 LM014699
6 · The paper itself

Abstract

backgroundNew therapeutics in oncology have presented challenges to existing paradigms and trial designs in all phases of drug development. As a motivating example, we considered an ongoing phase II trial planned to evaluate the combination of a MET inhibitor and an anti-PD-L1 immunotherapy to treat advanced oesogastric carcinoma. The objective of the paper was to exemplify the planning of an adaptive phase II trial with novel anti-cancer agents, including prolonged observation windows and joint sequential evaluation of efficacy and toxicity.

methodsWe considered various candidate designs and computed decision rules assuming correlations between efficacy and toxicity. Simulations were conducted to evaluate the operating characteristics of all designs.

resultsDesign approaches allowing continuous accrual, such as the time-to-event Bayesian Optimal Phase II design (TOP), showed good operating characteristics while ensuring a reduced trial duration. All designs were sensitive to the specification of the correlation between efficacy and toxicity during planning, but TOP can take that correlation into account more easily.

conclusionsWhile specifying design working hypotheses requires caution, Bayesian approaches such as the TOP design had desirable operating characteristics and allowed incorporating concomittant information, such as toxicity data from concomitant observations in another relevant patient population (e.g., defined by mutational status).

Indexed as

Bayes TheoremResearch DesignAntineoplastic AgentsClinical Trials, Phase II as TopicComputer SimulationDigestive System NeoplasmsHumansImmunotherapyAntineoplastic AgentsAdaptive designBayesianOncologyPhase II

Identifiers

PMID39030498
PMCPMC11526600

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Registered trials

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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.