Off-label high-efficacy immunotherapies, like rituximab, natalizumab, and fingolimod, had comparable efficacy to on-label prescription drugs for progressive forms of multiple sclerosis (MS), a network meta-analysis suggests.

The results showed the median Expanded Disability Severity Scale (EDSS) risk of progression was 35% in the placebo group and 30% in the immunotherapy group at 2 years, reported Guilherme Diogo Silva, of the University of São Paulo in Brazil, and colleagues.

The results were published in Acta Neurologica Scandinavica

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Our findings provide reassurance to many neurologists in clinical practice in maintaining MS patients on treatment with high-efficacy [disease-modifying drugs], including off-label drugs, even when they evolve to secondary progressive phenotypes of the disease,” the study authors wrote.

Read more about disease-modifying treatments for MS

Not all patients with MS have access to high-efficacy on-label drugs used to treat the progressive forms of the disease. In order to assess whether the prescription of other off-label disease-modifying drugs could improve the access of MS patients to immunotherapy, a team of researchers from Brazil compared the effect of on-and off-label high-efficacy drugs on the progression of disability in patients with progressive MS.

The analysis included 5 studies involving 4526 patients. There were 1872 patients in the placebo cohort and 2654 in the immunotherapy cohort. They used the Scopus, Embase, Cochrane Central, and MEDLINE databases to identify these studies.

The results also showed that off-label drugs had similar efficacy compared to ocrelizumab and siponimod, the 2 on-label drugs that are used to treat progressive MS.

“The use of fingolimod, rituximab, or natalizumab may be a strategy that reduces costs and improves access to immunotherapy for patients,” the study team concluded.


Silva GD, Castrillo BB, Apóstolos-Pereira SL, Callegaro D. Is there a role for off-label high-efficacy disease-modifying drugs in progressive multiple sclerosis? a network meta-analysis. Acta Neurol Scand. Published online September 2, 2022. doi:10.1111/ane.13697