Individual treatment effect prediction for amyotrophic lateral sclerosis patients

Statistical Methods in Medical Research
Heidi SeiboldTorsten Hothorn

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...Continue Reading

References

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Citations

Aug 25, 2017·Statistical Methods in Medical Research·Hoora MoradianFrançois Bellavance
May 31, 2019·Multiple Sclerosis : Clinical and Laboratory Research·Fabio PellegriniMaria Pia Sormani
Aug 20, 2019·Statistics in Medicine·Aniek Sies, Iven Van Mechelen
Dec 22, 2019·Statistics in Medicine·Julia KrzykallaAnnette Kopp-Schneider
Jun 23, 2020·Expert Review of Neurotherapeutics·Pamela A McCombeRobert D Henderson
Aug 28, 2020·Neurodegenerative Disease Management·Yuji Saitoh, Yuji Takahashi
Mar 16, 2019·Frontiers in Neuroscience·Vincent GrollemundPeter Bede
Aug 9, 2020·The International Journal of Biostatistics·Muriel Buri, Torsten Hothorn
Aug 12, 2021·Molecular Neurodegeneration·Laura PasettoValentina Bonetto
Sep 28, 2021·Statistical Methods in Medical Research·Chi ChangUNKNOWN Pooled Resource Open-Access ALS Clinical Trials Consortium

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Software Mentioned

R partykit
survival
eha
R
R system for computing
sandwich
ggplot2

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