Evaluation of longitudinal surrogate markers.
functional data
kernel smoothing
longitudinal data
nonparametric analysis
surrogate markers
Journal
Biometrics
ISSN: 1541-0420
Titre abrégé: Biometrics
Pays: United States
ID NLM: 0370625
Informations de publication
Date de publication:
06 2021
06 2021
Historique:
received:
27
09
2019
accepted:
05
05
2020
pubmed:
9
6
2020
medline:
26
10
2021
entrez:
8
6
2020
Statut:
ppublish
Résumé
The use of surrogate markers to examine the effectiveness of a treatment has the potential to decrease study length and identify effective treatments more quickly. Most available methods to investigate the usefulness of a surrogate marker involve restrictive parametric assumptions and tend to focus on settings where the surrogate is measured at a single point in time. However, in many clinical settings, the potential surrogate marker is often measured repeatedly over time, and thus, the surrogate marker information is a trajectory of measurements. In addition, it is often difficult in practice to correctly specify the relationship between a treatment, primary outcome, and surrogate marker trajectory. In this paper, we propose a model-free definition for the proportion of the treatment effect on the primary outcome that is explained by the treatment effect on the longitudinal surrogate markers. We propose three novel flexible methods to estimate this proportion, develop the asymptotic properties of our estimators, and investigate the robustness of the estimators under multiple settings via a simulation study. We apply our proposed procedures to an AIDS clinical trial dataset to examine a trajectory of CD4 counts as a potential surrogate.
Identifiants
pubmed: 32506496
doi: 10.1111/biom.13310
pmc: PMC8015060
mid: NIHMS1669906
doi:
Substances chimiques
Biomarkers
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
477-489Subventions
Organisme : NIDDK NIH HHS
ID : R01 DK118354
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL088589
Pays : United States
Informations de copyright
© 2020 The International Biometric Society.
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