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Individual treatment effect prediction for ALS patients


Seibold, Heidi; Zeileis, Achim; Hothorn, Torsten (2017). Individual treatment effect prediction for ALS patients. Statistical Methods in Medical Research:Epub ahead of print.

Abstract

A treatment for a complicated disease might be helpful for some but not all patients, which makes predicting the treatment effect for new patients important yet challenging. Here we develop a method for predicting the treatment effect based on patient characteristics and use it for predicting the effect of the only drug (Riluzole) approved for treating amyotrophic lateral sclerosis. Our proposed method of model-based random forests detects similarities in the treatment effect among patients and on this basis computes personalised models for new patients. The entire procedure focuses on a base model, which usually contains the treatment indicator as a single covariate and takes the survival time or a health or treatment success measurement as primary outcome. This base model is used both to grow the model-based trees within the forest, in which the patient characteristics that interact with the treatment are split variables, and to compute the personalised models, in which the similarity measurements enter as weights. We applied the personalised models using data from several clinical trials for amyotrophic lateral sclerosis from the Pooled Resource Open–Access Clinical Trials database. Our results indicate that some amyotrophic lateral sclerosis patients benefit more from the drug Riluzole than others. Our method allows gradually shifting from stratified medicine to personalised medicine and can also be used in assessing the treatment effect for other diseases studied in a clinical trial.

Abstract

A treatment for a complicated disease might be helpful for some but not all patients, which makes predicting the treatment effect for new patients important yet challenging. Here we develop a method for predicting the treatment effect based on patient characteristics and use it for predicting the effect of the only drug (Riluzole) approved for treating amyotrophic lateral sclerosis. Our proposed method of model-based random forests detects similarities in the treatment effect among patients and on this basis computes personalised models for new patients. The entire procedure focuses on a base model, which usually contains the treatment indicator as a single covariate and takes the survival time or a health or treatment success measurement as primary outcome. This base model is used both to grow the model-based trees within the forest, in which the patient characteristics that interact with the treatment are split variables, and to compute the personalised models, in which the similarity measurements enter as weights. We applied the personalised models using data from several clinical trials for amyotrophic lateral sclerosis from the Pooled Resource Open–Access Clinical Trials database. Our results indicate that some amyotrophic lateral sclerosis patients benefit more from the drug Riluzole than others. Our method allows gradually shifting from stratified medicine to personalised medicine and can also be used in assessing the treatment effect for other diseases studied in a clinical trial.

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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > Epidemiology, Biostatistics and Prevention Institute (EBPI)
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:2017
Deposited On:12 Jan 2017 09:39
Last Modified:06 Mar 2017 11:41
Publisher:Sage Publications Ltd.
ISSN:0962-2802
Publisher DOI:https://doi.org/10.1177/0962280217693034
Related URLs:https://arxiv.org/abs/1604.08720

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