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Related papers: Individual Treatment Effect Prediction for ALS Pat…

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The estimation of heterogeneous treatment effects (HTEs) has attracted considerable interest in many disciplines, most prominently in medicine and economics. Contemporary research has so far primarily focused on continuous and binary…

Methodology · Statistics 2022-10-07 Susanne Dandl , Andreas Bender , Torsten Hothorn

Amyotrophic lateral sclerosis (ALS) is a degenerative disorder of the motor neurons that causes progressive paralysis in patients. Current treatment options aim to prolong survival and improve quality of life. However, due to the…

Quantitative Methods · Quantitative Biology 2025-11-20 Christian Marius Lillelund , Sanjay Kalra , Russell Greiner

The identification of patient subgroups with differential treatment effects is the first step towards individualised treatments. A current draft guideline by the EMA discusses potentials and problems in subgroup analyses and formulated…

Methodology · Statistics 2020-01-22 Heidi Seibold , Achim Zeileis , Torsten Hothorn

Today, treatment effect estimation at the individual level is a vital problem in many areas of science and business. For example, in marketing, estimates of the treatment effect are used to select the most efficient promo-mechanics; in…

Machine Learning · Computer Science 2019-12-04 Aleksey Buzmakov , Daria Semenova , Maria Temirkaeva

Objective: To compare different risk-based methods for optimal prediction of treatment effects. Methods: We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk (PI), the shape…

A new matching method is proposed for the estimation of the average treatment effect of social policy interventions (e.g., training programs or health care measures). Given an outcome variable, a treatment and a set of pre-treatment…

Statistics Theory · Mathematics 2007-06-13 Stefano Iacus , Giuseppe Porro

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

Methodology · Statistics 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

Estimation of individual treatment effect in observational data is complicated due to the challenges of confounding and selection bias. A useful inferential framework to address this is the counterfactual (potential outcomes) model which…

Machine Learning · Statistics 2017-01-23 Min Lu , Saad Sadiq , Daniel J. Feaster , Hemant Ishwaran

The causal effect of a treatment can vary from person to person based on their individual characteristics and predispositions. Mining for patterns of individual-level effect differences, a problem known as heterogeneous treatment effect…

Machine Learning · Computer Science 2019-09-04 Christopher Tran , Elena Zheleva

Objective: In randomized clinical trials, prediction models can be used to explore the relationships between patients' variables (e.g., clinical, pathological, or lifestyle variables, and also biomarker or genomic data) and treatment effect…

Quantitative Methods · Quantitative Biology 2026-02-03 Elvire Roblin , Paul-Henry Cournède , Stefan Michiels

Survival prediction with small sets of features is a highly relevant topic for decision-making in clinical practice. I describe a method for predicting survival of amyotrophic lateral sclerosis (ALS) patients that was developed as a…

Applications · Statistics 2016-12-05 Christoph Kurz

We investigate the effect of the proportional hazards assumption on prognostic and predictive models of the survival time of patients suffering from amyotrophic lateral sclerosis (ALS). We theoretically compare the underlying model…

Methodology · Statistics 2019-10-22 Natalia Korepanova , Heidi Seibold , Verena Steffen , Torsten Hothorn

One size fits all approaches to medicine have become a thing of the past as the understanding of individual differences grows. The paper introduces a test for the presence of heterogeneity in treatment effects in a clinical trial.…

Accurately estimating personalized treatment effects within a study site (e.g., a hospital) has been challenging due to limited sample size. Furthermore, privacy considerations and lack of resources prevent a site from leveraging…

Machine Learning · Statistics 2022-06-17 Xiaoqing Tan , Chung-Chou H. Chang , Ling Zhou , Lu Tang

Treatment effect heterogeneity occurs when individual characteristics influence the effect of a treatment. We propose a novel approach that combines prognostic score matching and conditional inference trees to characterize effect…

Assessing heterogeneous treatment effects has become a growing interest in advancing precision medicine. Individualized treatment effects (ITE) play a critical role in such an endeavor. Concerning experimental data collected from randomized…

Machine Learning · Statistics 2018-07-23 Xiaogang Su , Annette T. Peña , Lei Liu , Richard A. Levine

This work demonstrates how mixed effects random forests enable accurate predictions of depression severity using multimodal physiological and digital activity data collected from an 8-week study involving 31 patients with major depressive…

Machine Learning · Computer Science 2023-01-25 Robert A. Lewis , Asma Ghandeharioun , Szymon Fedor , Paola Pedrelli , Rosalind Picard , David Mischoulon

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

Many scientific and engineering challenges -- ranging from personalized medicine to customized marketing recommendations -- require an understanding of treatment effect heterogeneity. In this paper, we develop a non-parametric causal forest…

Methodology · Statistics 2017-07-11 Stefan Wager , Susan Athey

We conducted a proof-of-concept evaluation of individualized treatment effect (ITE) estimation using survival data from a randomized trial of 475 men with advanced prostate cancer treated with high- versus low-dose diethylstilbestrol (DES).…

Applications · Statistics 2025-08-26 J. T. Korley
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