Efficacy outcomes reported in trials of multiple sclerosis: A systematic scoping review
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Magda Laíse Oliveira1, Rosa Camila Lucchetta2, Aline de Fátima Bonetti2
1Graduate Program in Pharmaceutical Assistance, Federal University of Paraná, Curitiba, Paraná, Brazil.
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.
Insights
Clinical efficacy outcomes for multiple sclerosis treatments lack consensus. This review found 77 different outcomes and 36 definitions for relapse, highlighting the need for standardized measures in relapsing forms of multiple sclerosis research.
Area of Science:
- Neurology
- Clinical Trials
- Evidence-Based Medicine
Background:
- Disease-modifying therapies (DMT) are crucial for managing relapsing forms of multiple sclerosis (RMS).
- Randomized Controlled Trials (RCTs) are key in evaluating DMT efficacy.
- Standardized outcome measures are needed for consistent evaluation of DMT in RMS.
Purpose of the Study:
- To systematically map and analyze clinical efficacy outcome measures used in RCTs for DMT in adult patients with RMS.
- To identify the range and definitions of reported outcomes, including composite measures and relapse definitions.
Main Methods:
- A systematic scoping review was conducted, searching PubMed, Scopus, and The Cochrane Controlled Register of Trials.
- Included RCTs assessing DMT efficacy in adult patients with RMS.
- Descriptive-quantitative analysis of identified clinical efficacy outcomes and their definitions.
Main Results:
- 226 RCTs were included, with 89% reporting clinical efficacy outcomes.
- 77 distinct efficacy outcomes were identified, alongside five composite outcomes.
- Significant heterogeneity was found, including 36 different definitions for 'relapse' and six definitions for 'no evidence of radiological and clinic disease activity'.
Conclusions:
- A lack of consensus exists regarding clinical efficacy outcome measures in RCTs for RMS.
- Wide heterogeneity in outcome definitions necessitates the development of a standardized core outcome set.
- This research provides a foundation for establishing a core outcome set for clinical efficacy in adult RMS.
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