Evidence map›Paper›PMID 39468894›Full record

ArticleStatistics in medicine2024

Estimands and Cumulative Incidence Function Regression in Clinical Trials: Some New Results on Interpretability and Robustness.

Alexandra Bühler, Richard J Cook, Jerald F Lawless

Abstract read
In one paragraph

Article in Statistics in medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. Estimands in clinical trials of complex disease processes.Clinical trials (London, England) · 2024
    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

3 authors.

Alexandra BühlerDepartment of Statistics and Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada.ORCID 0000-0002-0872-0374
Richard J CookDepartment of Statistics and Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada.ORCID 0000-0002-1414-4908
Jerald F LawlessDepartment of Statistics and Actuarial Science, University of Waterloo, Waterloo, Ontario, Canada.ORCID 0000-0002-3192-0470

Funding

Natural Sciences and Engineering Research Council of Canada RGPIN-2017-04055Natural Sciences and Engineering Research Council of Canada RGPIN-2017-04207
6 · The paper itself

Abstract

Regression analyses based on transformations of cumulative incidence functions are often adopted when modeling and testing for treatment effects in clinical trial settings involving competing and semi-competing risks. Common frameworks include the Fine-Gray model and models based on direct binomial regression. Using large sample theory we derive the limiting values of treatment effect estimators based on such models when the data are generated according to multiplicative intensity-based models, and show that the estimand is sensitive to several process features. The rejection rates of hypothesis tests based on cumulative incidence function regression models are also examined for null hypotheses of different types, based on which a robustness property is established. In such settings supportive secondary analyses of treatment effects are essential to ensure a full understanding of the nature of treatment effects. An application to a palliative study of individuals with breast cancer metastatic to bone is provided for illustration.

Indexed as

Breast NeoplasmsModels, StatisticalBone NeoplasmsClinical Trials as TopicData Interpretation, StatisticalFemaleHumansIncidenceRegression Analysisclinical trialscompeting risksestimandsgeneralized linear modelslarge sample resultsrobustnesstesting

Identifiers

PMID39468894
PMCPMC11589047

What OpenQuestion holds

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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.