Evidence map›Paper›PMID 39114952›Full record

ArticleClinical trials (London, England)2024

Inferences for the distribution of the duration of response in a comparative clinical study.

Ying Cui, Bo Huang, Lu Mao, Hajime Uno, Lee-Jen Wei, Lu Tian

Abstract read
In one paragraph

Article in Clinical trials (London, England), 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. Review
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

6 authors.

Ying CuiDepartment of Biomedical Data Science, Stanford University, Stanford, CA, USA.ORCID 0000-0002-3697-5155
Bo HuangPfizer Research & Development, Pfizer Inc, Groton, CT, USA.
Lu MaoDepartment of Biostatistics and Medical Informatics, School of Medicine and Public Health, University of Wisconsin-Madison, Madison, WI, USA.ORCID 0000-0002-8626-9822
Hajime UnoDepartment of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.ORCID 0000-0003-0622-8471
Lee-Jen WeiDepartment of Biostatistics, Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, USA.ORCID 0000-0002-8582-920X
Lu TianDepartment of Biomedical Data Science, Stanford University, Stanford, CA, USA.

Funding

Statistical Methods for Optimizing Personalized Treatment SelectionR01HL089778 · NHLBI · STANFORD UNIVERSITY · PI LU TIAN · 2008 to 2026
$4.8M
Novel Statistical Methods for Complex Time-to-Event Data in Cardiovascular Clinical TrialsR01HL149875 · NHLBI · UNIVERSITY OF WISCONSIN-MADISON · PI Lu Mao · 2020 to 2026
$2.4M
Survival Data Analysis using general censoring-free incident rateR01GM152499 · NIGMS · DANA-FARBER CANCER INST · PI Hajime Uno · 2024 to 2026
$1.0M
NHLBI NIH HHS R01 HL089778NHLBI NIH HHS R01 HL149875NIGMS NIH HHS R01 GM152499
6 · The paper itself

Abstract

Duration of response is an important endpoint used in drug development. Prolonged duration for response is often viewed as an early indication of treatment efficacy. However, there are numerous difficulties in studying the distribution of duration of response based on observed data subject to right censoring in practice. The most important obstacle is that the distribution of the duration of response is in general not identifiable in the presence of censoring due to the simple fact that there is no information on the joint distribution of time to response and time to progression beyond the largest follow-up time. In this article, we introduce the restricted duration of response as a replacement of the conventional duration of response. The distribution of restricted duration of response is estimable and we have proposed several nonparametric estimators in this article. The corresponding inference procedure and additional downstream analysis have been developed. Extensive numerical simulations have been conducted to examine the finite sample performance of the proposed estimators. It appears that a new regression-based two-step estimator for the survival function of the restricted duration of response tends to have a robust and superior performance, and we recommend its use in practice. A real data example from oncology has been used to illustrate the analysis for restricted duration of response.

Indexed as

Computer SimulationData Interpretation, StatisticalEndpoint DeterminationHumansModels, StatisticalResearch DesignStatistics, NonparametricTime FactorsTreatment Outcomecomparative clinical studyDuration of responseestimation and inferencenonparametric estimatorsrestricted duration of response

Identifiers

PMID39114952
PMCPMC12113349

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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.