Vinciguerra Claudia, D'Amico Anna, Bevilacqua Liliana, Rini Nicasio, D'Apolito Maria, Liberatoscioli Eliana, Monastero Roberto, Barone Paolo, Brighina Filippo, Di Muzio Antonio, Di Stefano Vincenzo
Neurology Unit, Department of Medicine, Surgery and Dentistry "Scuola Medica Salernitana", University Hospital San Giovanni di Dio e Ruggi D'Aragona, 84131 Salerno, Italy.
Department of Biomedicine, Neuroscience and Advanced Diagnostics (BIND), University of Palermo, 90127 Palermo, Italy.
Brain Sci. 2024 Jul 31;14(8):774. doi: 10.3390/brainsci14080774.
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.
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.
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 ( < 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.
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.
重症肌无力（MG）是一种自身免疫性疾病，其特征是由于针对神经肌肉接头蛋白的自身抗体导致肌肉无力波动。霉酚酸酯（MMF）作为一种免疫抑制疗法，与其他治疗方法相比，在治疗MG方面显示出副作用较少的潜力。本研究旨在评估MMF在现实生活中的多中心环境下对MG患者的有效性和安全性。
对2011年1月至2024年2月在意大利三个中心接受治疗的全身型MG患者进行回顾性队列研究，这些患者对硫唑嘌呤（AZA）难治，单独使用MMF或联合使用类固醇进行治疗。在基线、6个月、12个月、18个月和24个月时，使用美国重症肌无力基金会（MGFA）分类、MG综合评分（MGCS）和MG日常生活活动（MGADL）评分对患者进行评估。统计分析包括Spearman相关性分析、Friedman检验和方差分析。
共纳入32例患者（13例男性，平均年龄66.5±11.5岁）。在6个月和12个月时观察到MGADL和MGCS评分有显著改善（<0.001），并在24个月内持续改善。12%的患者报告有副作用。与硫唑嘌呤相比，MMF症状控制起效更快，在6个月内有显著改善。
最近一项研究发现，MMF和AZA在改善患者生活质量方面同样有效，但由于AZA比MMF有更严重的不良事件，因此建议使用较低剂量的AZA以减少不良事件同时保持疗效。相反，结果表明MMF在MG的长期管理中有效且耐受性良好，能更快地控制症状并具有良好的安全性。未来需要进行更大样本量的前瞻性研究来证实这些发现，并探索MMF治疗反应中的性别差异。
