Leflunomide  plus low-dose prednisone (0.25 mg/kg daily) could considerably improve the symptoms of myasthenia gravis (MG) in the long term, according to the results of a new retrospective study published in the journal Acta Neurologica Belgica. The treatment also seems to be well tolerated with few side effects, the researchers found.

It was already known that leflunomide  plus low-dose prednisone is able to rapidly improve MG symptoms, they noted.

Read more about the symptoms of MG

The aim of the present study was to analyze the long-term safety and efficacy of the treatment regimen.

A team of researchers from China conducted a retrospective cohort study in 103 patients with MG who were continuously treated with leflunomide  plus low-dose prednisone for at least 1 year between 2012 and 2020.

The study team reported that effective treatment was achieved in the majority of the patients (58.3%) after 1 month. After 1 year of treatment, efficacy was achieved in 88.4% of the patients with 61.2% exhibiting only minimal manifestations of the disease. 

The average MG activities of daily living (MG-ADL) score, which is a measure of the impact of MG on daily functions, decreased from 6 to 1. The average quantitative MG (QMG) scale scores also went down from 10 to 4. 

These decreases were more pronounced in patients with generalized MG compared to patients with ocular MG. 

Patients who underwent a thymectomy had a smaller decrease in MG-ADL and QMG scores compared to those who did not. 

The researchers reported 16 adverse effects associated with the treatment but said that none of them were severe.

Leflunomide is an isoxazole derivative that inhibits the proliferation of T and B cells by acting on the dihydroorotate synthase enzyme, which is essential for pyrimidine synthesis. It has widely been used to treat other autoimmune diseases such as rheumatoid arthritis and lupus nephritis.


Qiu L, Chen P, Ou C, et al. Long-term efficacy and safety of leflunomide combined with low-dose prednisone in [the] treatment of myasthenia gravis: a retrospective study. Acta Neurol Belg. Published online September 1, 2023. doi:10.1007/s13760-023-02367-y