Single Index Methods for Estimation and Evaluation of Marker-Guided Treatment Rules Based on Multivariate Marker Panels

• Activity Number: 410
• Type: Topic Contributed
• Date/Time: Tuesday, August 5, 2014 : 2:00 PM to 3:50 PM
• Sponsor: WNAR
• Author(s): Veronika Skrivankova*+ and Patrick Heagerty
• Companies: University of Washington and University of Washington
• Keywords: marker-guided treatment ; evaluation of treatment rules ; treatment benefit score ; single index ; cross-validation

Abstract

Clinical practice may be enhanced by use of person-level information that could guide treatment choice and lead to both better outcomes for treated individuals and for the population. The scientific challenge is to identify those factors that can be used to target treatment, and to accurately quantify the expected treatment benefit as a function of candidate markers. The proposed research develops statistical methodology for the generation and evaluation of a single index score, that estimates the expected treatment benefit associated with patient characteristics as a linear combination of the markers. Our methods specifically decouple the model used to generate a treatment benefit score from non-parametric methods that are adopted to evaluate the score. Cross-validation methods ensure honest evaluation of the score performance at the population level if it was used to guide treatment. We also show that the treatment benefit score can be used for selecting a subset of patients with enriched treatment response. The methods are illustrated on data from a randomized trial of steroid injections where baseline clinical and imaging data are candidate measures for guiding therapy.