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Assessing heterogeneity of treatment effect in multiple sclerosis trials.


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.

SUBMITTER: Sormani MP 

PROVIDER: S-EPMC10413777 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature

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Assessing heterogeneity of treatment effect in multiple sclerosis trials.

Sormani Maria Pia MP   Chataway Jeremy J   Kent David M DM   Marrie Ruth Ann RA  

Multiple sclerosis (Houndmills, Basingstoke, England) 20230801 9


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 appro  ...[more]

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