A two-stage prediction model for heterogeneous effects of treatments.

Chalkou, Konstantina; Steyerberg, Ewout; Egger, Matthias; Manca, Andrea; Pellegrini, Fabio; Salanti, Georgia (2021). A two-stage prediction model for heterogeneous effects of treatments. Statistics in medicine, 40(20), pp. 4362-4375. Wiley-Blackwell 10.1002/sim.9034

Preview

Text Chalkou_StatMed_2021.pdf - Published Version Available under License Creative Commons: Attribution (CC-BY). Download (1MB) | Preview

Treatment effects vary across different patients, and estimation of this variability is essential for clinical decision-making. We aimed to develop a model estimating the benefit of alternative treatment options for individual patients, extending a risk modeling approach in a network meta-analysis framework. We propose a two-stage prediction model for heterogeneous treatment effects by combining prognosis research and network meta-analysis methods where individual patient data are available. In the first stage, a prognostic model to predict the baseline risk of the outcome. In the second stage, we use the baseline risk score from the first stage as a single prognostic factor and effect modifier in a network meta-regression model. We apply the approach to a network meta-analysis of three randomized clinical trials comparing the relapses in Natalizumab, Glatiramer Acetate, and Dimethyl Fumarate, including 3590 patients diagnosed with relapsing-remitting multiple sclerosis. We find that the baseline risk score modifies the relative and absolute treatment effects. Several patient characteristics, such as age and disability status, impact the baseline risk of relapse, which in turn moderates the benefit expected for each of the treatments. For high-risk patients, the treatment that minimizes the risk of relapse in 2 years is Natalizumab, whereas Dimethyl Fumarate might be a better option for low-risk patients. Our approach can be easily extended to all outcomes of interest and has the potential to inform a personalized treatment approach.

Interest & Impact

Downloads

213 since deposited on 03 Jun 2021
114 in the past 12 months

Citations

5 Citations in Web of Science ®
6 Citations in Scopus

Search

in Google Scholar™

Services

Actions (login required)

Edit item

Actions (login required)

Edit item