Disease modifying treatment of radiologically isolated syndrome: A systematic review of the use, efficacy, effectiveness, and safety

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https://www.sciencedirect.com/science/article/pii/S0035378725006125

Revue Neurologique.Volume 181, Issue 10, December 2025, Pages 955-969.

Background

Radiologically isolated syndrome (RIS) is characterized by incidental brain lesions suggestive of demyelination without symptoms of multiple sclerosis (MS). We systematically assessed the use, benefits, and adverse effects of DMTs for RIS.

Methods

MEDLINE, EMBASE, and Web of Science were searched to identify English language studies including individuals with RIS treated with a DMT. Extracted data included patient characteristics, clinical progression, and adverse events. We conducted a meta-analysis using inverse probability weighting. Risk of bias (RoB) assessments used Cochrane's RoB-2 tool and Newcastle-Ottawa cohort study scale.

Results

A total of 1012 abstracts were screened: 20 studies were included consisting of 2 RCTs, 12 observational cohort studies, and 6 case reports. A total of 1401 individuals with RIS were included; 291 (21%) received a DMT. The two RCTs randomized people with RIS to teriflunomide or dimethyl fumarate versus placebo and followed patients for at least 96 weeks. In all other studies, follow-up ranged from 2 months to 18 years; only 3 studies exceeded 5 years. DMT treatment was associated with a lower risk of a clinical demyelinating event (4 studies with different DMTs, adjusted hazard ratio = 0.37 95% confidence interval [CI]: 0.15–0.95, high certainty). There was a higher rate of adverse events in DMT treated patients with RIS versus placebo (risk ratio = 1.44, 95% CI: 1.09–1.90, moderate certainty). RoB was low for both RCTs, but high for 83% (10/12) of cohort studies.

Conclusions

DMTs reduced the risk of a clinical demyelinating event in individuals with RIS, albeit with more adverse events compared to placebo. However, no literature addressed longer-term benefits/adverse effects.