Efficacy outcomes reported in trials of multiple sclerosis: A systematic scoping review

Abstract

Background: This study aimed to map the outcome measures of clinical efficacy reported in Randomized Controlled Trials (RCT) to evaluate disease-modifying therapies (DMT) in patients with relapsing forms of multiple sclerosis (RMS).

Methods: A systematic scoping review was performed to identify RCT that assessed the efficacy of DMT in adult patients with RMS. Searches were conducted in PubMed, Scopus, and The Cochrane Controlled Register of Trials and complemented by manual search. A descriptive-quantitative analysis of the clinical efficacy outcomes with their respective definitions was performed.

Results: Of the 5,476 records identified, 226 were included. Among the included studies, 89% reported clinical efficacy outcomes, with 77 different outcomes identified, including five composite outcomes. A total of 36 different definitions for 'relapse' were identified. 'Annualized relapse rate' was the most prevalent single outcome (n = 56 studies). At the same time, the 'Proportion of patients with no evidence of radiological and clinic disease activity' was the most prevalent composite outcome (n = 14 studies) although with six different definitions.

Conclusions: An absence of consensus on the clinical efficacy outcomes reported in RCT associated with a wide heterogeneity of definitions were identified. The mapped results of this research can be used as a basis for the definition of a core outcome set for clinical efficacy outcomes in adults with RMS.

Keywords: Efficacy; Exposure; Measures of association; Multiple sclerosis; Outcome research; Relapsing-remitting multiple sclerosis; Risk or outcome.