ArticleStatistics in medicine2025
Specification of Estimands for Complex Disease Processes Using Multistate Models and Utility Functions.
Article in Statistics in medicine, 2025. 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
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.
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.
- Specification of Estimands for Complex Disease Processes Using Multistate Models and Utility Functions.Statistics in medicine · 2025Article
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Authors and funding
3 authors.
Funding
Abstract
In complex diseases, individuals are often at risk of several types of possibly semi-competing events and may experience recurrent symptomatic episodes. This complex disease course makes it challenging to define target estimands for clinical trials. While composite endpoints are routinely adopted, recent innovations involving the win ratio and other methods based on ranking the disease course have received considerable attention. We emphasize the usefulness of multistate models for addressing challenges arising in complex diseases, along with the simplicity and interpretability that come from defining utilities to synthesize evidence of treatment effects on different aspects of the disease process. Robust variance estimation based on the infinitesimal jackknife means that such methods can be used as the basis of primary analyses of clinical trials. We illustrate the use of utilities for the assessment of bleeding outcomes in a trial of cancer patients with thrombocytopenia.
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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.