Work

Treatment selections using risk-benefit profiles based on data from comparative randomized clinical trials with multiple endpoints

Year: 2014

Type: article

Abstract: In a typical randomized clinical study to compare a new treatment with a control, oftentimes each study subject may experience any of several distinct outcomes during the study period, which collectively define the "risk-benefit" profile. To assess the effect of treatment, it is desirable to utilize the entirety of such outcome information. The times to these events, however, may not be observed completely due to, for example, competing risks or administrative censoring. The standard analyses based on the time to the first event, or individual component analyses with respect to each event time, are not ideal. In this paper, we classify each patient's risk-benefit profile, by considering all event times during follow-up, into several clinically meaningful ordinal categories. We first show how to make inferences for the treatment difference in a two-sample setting where categorical data are incomplete due to censoring. We then present a systematic procedure to identify patients who would benefit from a specific treatment using baseline covariate information. To obtain a valid and efficient system for personalized medicine, we utilize a cross-validation method for model building and evaluation and then make inferences using the final selected prediction procedure with an independent data set. The proposal is illustrated with the data from a clinical trial to evaluate a beta-blocker for treating chronic heart failure patients.

Source: Biostatistics

DOI: 10.1093/biostatistics/kxu037

Authors: B. Claggett, L. Tian, D. Castagno, L.-J. Wei

Institutions: Harvard University, Stanford Medicine, Stanford University, University of Turin

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

SDG: Good health and well-being

Open Access status: bronze