Evaluating epoetin dosing strategies using observational longitudinal data

DOI10.1214/14-AOAS774MaRDI QIDQ2258584zbMATH OpenOpenAlexFDO

Authors Cecilia A. Cotton, Patrick J. Heagerty

Publication date 26 February 2015

Published in The Annals of Applied Statistics (Search for Journal in Brave)

Full work available at URL https://arxiv.org/abs/1502.00770, https://projecteuclid.org/euclid.aoas/1419001747

zbMATH Keywords

marginal structural modelsobservational studiessurvival analysis

Mathematics Subject Classification ID

Estimation in survival analysis and censored data (62N02) Applications of statistics to biology and medical sciences; meta analysis (62P10)

Abstract: Epoetin is commonly used to treat anemia in chronic kidney disease and End Stage Renal Disease subjects undergoing dialysis, however, there is considerable uncertainty about what level of hemoglobin or hematocrit should be targeted in these subjects. In order to address this question, we treat epoetin dosing guidelines as a type of dynamic treatment regimen. Specifically, we present a methodology for comparing the effects of alternative treatment regimens on survival using observational data. In randomized trials patients can be assigned to follow a specific management guideline, but in observational studies subjects can have treatment paths that appear to be adherent to multiple regimens at the same time. We present a cloning strategy in which each subject contributes follow-up data to each treatment regimen to which they are continuously adherent and artificially censored at first nonadherence. We detail an inverse probability weighted log-rank test with a valid asymptotic variance estimate that can be used to test survival distributions under two regimens. To compare multiple regimens, we propose several marginal structural Cox proportional hazards models with robust variance estimation to account for the creation of clones. The methods are illustrated through simulations and applied to an analysis comparing epoetin dosing regimens in a cohort of 33,873 adult hemodialysis patients from the United States Renal Data System.

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Cites work

• Analysis of Semiparametric Regression Models for Repeated Outcomes in the Presence of Missing Data
• Correcting for Noncompliance and Dependent Censoring in an AIDS Clinical Trial with Inverse Probability of Censoring Weighted (IPCW) Log‐Rank Tests
• Evaluating epoetin dosing strategies using observational longitudinal data
• scientific article; zbMATH DE number 124964 (Why is no real title available?)
• scientific article; zbMATH DE number 1487685 (Why is no real title available?)
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• Partly Conditional Survival Models for Longitudinal Data
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Cited in

(2)

• Personalized medicine -- optimized EPO dosing
• Evaluating epoetin dosing strategies using observational longitudinal data

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