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Azathioprine for people with multiple sclerosis.

Ben Ridley¹, Francesco Nonino¹, Elisa Baldin¹

¹IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy.

The Cochrane Database of Systematic Reviews

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Azathioprine may modestly reduce relapses in multiple sclerosis (MS) but has uncertain effects on disability and may increase serious adverse events. More research is needed due to low-certainty evidence.

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Area of Science:

Neurology
Immunology
Pharmacology

Background:

Multiple sclerosis (MS) is a chronic, inflammatory, demyelinating disease of the central nervous system affecting 2.8 million people globally.
MS predominantly impacts young women, with a mean age of diagnosis at 32 years, posing a significant health burden.
Azathioprine (AZA), used for autoimmune diseases, is an off-label treatment for MS, particularly where access to approved disease-modifying treatments (DMTs) is limited.

Conclusions:

Azathioprine may offer modest benefits for relapse frequency in relapsing MS, but with a potential increase in SAEs compared to interferon beta.
The evidence for azathioprine's effects on disability progression and short-term adverse events is very uncertain.
Certainty in the available evidence is low to very low, highlighting the need for further research, especially for progressive MS and long-term outcomes.