Highlights
- •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.
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
Abbreviations:
ARR (annualized relapse rate), COS (core outcome set), DMT (disease-modifying therapies), DPC12 (disability progression confirmed at 12 weeks), DPC24 (disability progression confirmed at 24 weeks), EDSS (expanded disability status scale), JBI (Joanna Briggs Institute), MS (multiple sclerosis), NEDA-C (proportion of patients with no evidence of clinic disease activity), NEDA-CR (proportion of patients with no evidence of radiological and clinic disease activity), RCT (randomized controlled trials), RFP (relapse free patient), RMS (relapsing forms of multiple sclerosis), RRMS (relapsing-remitting multiple sclerosis)To read this article in full you will need to make a payment
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Article info
Publication history
Published online: July 30, 2020
Accepted:
July 29,
2020
Received in revised form:
July 27,
2020
Received:
June 6,
2020
Identification
Copyright
© 2020 Elsevier B.V. All rights reserved.
