Statistical Methods to Evaluate Surrogate Markers.
Med Care
; 62(2): 102-108, 2024 Feb 01.
Article
em En
| MEDLINE
| ID: mdl-38079232
ABSTRACT
BACKGROUND:
There is tremendous interest in evaluating surrogate markers given their potential to decrease study time, costs, and patient burden.OBJECTIVES:
The purpose of this statistical workshop article is to describe and illustrate how to evaluate a surrogate marker of interest using the proportion of treatment effect (PTE) explained as a measure of the quality of the surrogate marker for (1) a setting with a general fully observed primary outcome (eg, biopsy score); and (2) a setting with a time-to-event primary outcome which may be censored due to study termination or early drop out (eg, time to diabetes).METHODS:
The methods are motivated by 2 randomized trials, one among children with nonalcoholic fatty liver disease where the primary outcome was a change in biopsy score (general outcome) and another study among adults at high risk for Type 2 diabetes where the primary outcome was time to diabetes (time-to-event outcome). The methods are illustrated using the Rsurrogate package with a detailed R code provided.RESULTS:
In the biopsy score outcome setting, the estimated PTE of the examined surrogate marker was 0.182 (95% confidence interval [CI] 0.121, 0.240), that is, the surrogate explained only 18.2% of the treatment effect on the biopsy score. In the diabetes setting, the estimated PTE of the surrogate marker was 0.596 (95% CI 0.404, 0.760), that is, the surrogate explained 59.6% of the treatment effect on diabetes incidence.CONCLUSIONS:
This statistical workshop provides tools that will support future researchers in the evaluation of surrogate markers.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Diabetes Mellitus Tipo 2
Limite:
Child
/
Humans
Idioma:
En
Revista:
Med Care
Ano de publicação:
2024
Tipo de documento:
Article