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Assessing heterogeneity of treatment effect in multiple sclerosis trials

Sormani, Maria Pia; Chataway, Jeremy; Kent, David M; Marrie, Ruth Ann; (2023) Assessing heterogeneity of treatment effect in multiple sclerosis trials. Multiple Sclerosis Journal , 29 (9) pp. 1158-1161. 10.1177/13524585231189673. Green open access

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Abstract

Multiple sclerosis (MS) is heterogeneous with respect to outcomes, and evaluating possible heterogeneity of treatment effect (HTE) is of high interest. HTE is non-random variation in the magnitude of a treatment effect on a clinical outcome across levels of a covariate (i.e. a patient attribute or set of attributes). Multiple statistical techniques can evaluate HTE. The simplest but most bias-prone is conventional one variable-at-a-time subgroup analysis. Recently, multivariable predictive approaches have been promoted to provide more patient-centered results, by accounting for multiple relevant attributes simultaneously. We review approaches used to estimate HTE in clinical trials of MS.

Type: Article
Title: Assessing heterogeneity of treatment effect in multiple sclerosis trials
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1177/13524585231189673
Publisher version: https://doi.org/10.1177/13524585231189673
Language: English
Additional information: This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).
Keywords: Multiple sclerosis, clinical trial
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
URI: https://discovery.ucl.ac.uk/id/eprint/10176165
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