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. 2024 Jul 31;14(8):774.
doi: 10.3390/brainsci14080774.

Effectiveness and Safety of Mycophenolate Mophetil in Myasthenia Gravis: A Real-Life Multicenter Experience

Claudia Vinciguerra  1 , Anna D'Amico  2 , Liliana Bevilacqua  1 , Nicasio Rini  2 , Maria D'Apolito  3 , Eliana Liberatoscioli  3 , Roberto Monastero  2 , Paolo Barone  1 , Filippo Brighina  2 , Antonio Di Muzio  3 , Vincenzo Di Stefano  2
Affiliations

Effectiveness and Safety of Mycophenolate Mophetil in Myasthenia Gravis: A Real-Life Multicenter Experience

Claudia Vinciguerra et al. Brain Sci. .

Abstract

Background: Myasthenia gravis (MG) is an autoimmune disease characterized by fluctuating muscle weakness due to autoantibodies targeting neuromuscular junction proteins. Mycophenolate mofetil (MMF), an immunosuppressive therapy, has shown potential for managing MG with fewer side effects compared to other treatments. This study aims to evaluate the effectiveness and safety of MMF in MG patients in a real-life multicenter setting.

Methods: A retrospective cohort study was conducted on generalized MG patients, refractory to azathioprine (AZA) and treated with MMF alone or with steroids, at three Italian centers from January 2011 to February 2024. Patients were assessed using the Myasthenia Gravis Foundation of America (MGFA) classification, MG composite score (MGCS), and MG activity of daily living (MGADL) scores at baseline, 6, 12, 18, and 24 months. Statistical analyses included the Spearman correlation, the Friedman test, and ANOVA.

Results: Thirty-two patients were enrolled (13 males, mean age 66.5 ± 11.5 years). Significant improvements in MGADL and MGCS scores were observed at 6 and 12 months (p < 0.001), with continued improvement over 24 months. Side effects were reported in 12% of patients. MMF showed a faster onset of symptom control compared to azathioprine, with a significant improvement noted within 6 months.

Conclusions: A recent study found that MMF and AZA were equally effective in improving patients' quality of life, but because AZA had more serious adverse events than MMF, lower doses of AZA were therefore recommended to reduce the adverse events while maintaining efficacy. Conversely, results showed that MMF is effective and well-tolerated in the long-term management of MG, providing faster symptom control and a favorable safety profile. Future prospective studies with larger cohorts are needed to confirm these findings and explore sex differences in response to MMF treatment.

Keywords: autoimmune disease; immunosuppressive therapy; myasthenia gravis (MG); mycophenolate mofetil (MMF); neuromuscular junction; symptom control.

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Conflict of interest statement

The authors declare no conflicts of interest.

Figures

Figure 1
Figure 1
Bar diagram depicting the percentage distribution of comorbidities in the study population.
Figure 2
Figure 2
Illustrative graph of the trend improvement in the MGCS scale (A) and MGADL (B) at different time points (from baseline, 6, 12, 18, and 24 months).
Figure 3
Figure 3
The ANOVA test showing a significant correlation between the delta variations in MGCS scores at 12 months and sex.
Figure 4
Figure 4
The distribution of MGFA classification among patients at the start of MMF (left) and at last follow-up (right).
See this image and copyright information in PMC

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