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

•Definition of ‘relapse’ in RMS trials varies in studies.•Studies use a wide variety of outcomes to report clinical efficacy of the DMT used in RMS.•A map of outcomes used in literature can serve as a basis to a COS for clinical efficacy in RMS. This study aimed to map the outcome measures of clinic...

Full description

Saved in:

Bibliographic Details
Published in:	Multiple sclerosis and related disorders Vol. 45; p. 102435
Main Authors:	Oliveira, Magda Laíse, Lucchetta, Rosa Camila, Bonetti, Aline de Fátima, Fernandez-Llimós, Fernando, Becker, Jefferson, Gonçalves, Marcus Vinícius Magno, Tauil, Carlos Bernardo, Pontarolo, Roberto, Wiens, Astrid
Format:	Journal Article
Language:	English
Published:	Netherlands Elsevier B.V 01-10-2020
Subjects:	Adult Efficacy Exposure Humans Measures of association Multiple sclerosis Multiple Sclerosis - drug therapy Multiple Sclerosis, Relapsing-Remitting Outcome research Randomized Controlled Trials as Topic Recurrence Relapsing-remitting multiple sclerosis Risk or outcome Treatment Outcome RRMS ARR Multiple sclerosis RCT Measures of association COS MS NEDA-CR Efficacy Exposure DMT NEDA-C Outcome research JBI Risk or outcome Relapsing-remitting multiple sclerosis RMS DPC24 DPC12 RFP EDSS
Online Access:	Get full text
Tags:	Add Tag No Tags, Be the first to tag this record!

Description
Summary:	•Definition of ‘relapse’ in RMS trials varies in studies.•Studies use a wide variety of outcomes to report clinical efficacy of the DMT used in RMS.•A map of outcomes used in literature can serve as a basis to a COS for clinical efficacy in RMS. 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). 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. 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. 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.
Bibliography:	SourceType-Scholarly Journals-1 ObjectType-Feature-4 ObjectType-Undefined-1 content type line 23 ObjectType-Review-2 ObjectType-Article-3
ISSN:	2211-0348 2211-0356
DOI:	10.1016/j.msard.2020.102435