Evidence map›Paper›PMID 34047366›Full record

ArticleBiometrics2022

Sample size formula for general win ratio analysis.

Lu Mao, KyungMann Kim, Xinran Miao

Abstract read
In one paragraph

Article in Biometrics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing 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

11 citing papers in PubMed.

  1. Trial
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  3. Image optimization for low-doseEJNMMI physics · 2026
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  6. Review
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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.

Lu MaoDepartment of Biostatistics and Medical Informatics, University of Wisconsin-Madison, Madison, Wisconsin.ORCID 0000-0002-8626-9822
KyungMann KimDepartment of Biostatistics and Medical Informatics, University of Wisconsin-Madison, Madison, Wisconsin.ORCID 0000-0002-6726-003X
Xinran MiaoDepartment of Biostatistics and Medical Informatics, University of Wisconsin-Madison, Madison, Wisconsin.

Funding

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
NHLBI NIH HHS R01 HL149875
6 · The paper itself

Abstract

Originally proposed for the analysis of prioritized composite endpoints, the win ratio has now expanded into a broad class of methodology based on general pairwise comparisons. Complicated by the non-i.i.d. structure of the test statistic, however, sample size estimation for the win ratio has lagged behind. In this article, we develop general and easy-to-use formulas to calculate sample size for win ratio analysis of different outcome types. In a nonparametric setting, the null variance of the test statistic is derived using U-statistic theory in terms of a dispersion parameter called the standard rank deviation, an intrinsic characteristic of the null outcome distribution and the user-defined rule of comparison. The effect size can be hypothesized either on the original scale of the population win ratio, or on the scale of a "usual" effect size suited to the outcome type. The latter approach allows one to measure the effect size by, for example, odds/continuation ratio for totally/partially ordered outcomes and hazard ratios for composite time-to-event outcomes. Simulation studies show that the derived formulas provide accurate estimates for the required sample size across different settings. As illustration, real data from two clinical studies of hepatic and cardiovascular diseases are used as pilot data to calculate sample sizes for future trials.

Indexed as

Cardiovascular DiseasesComputer SimulationHumansOdds RatioProportional Hazards ModelsSample Sizecomposite outcomespartial orderpower analysisstandard rank deviationU-statisticWilcoxon-Mann-Whitney test

Identifiers

PMID34047366
PMCPMC8627514

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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.