Mantegazza Renato, Antonini Giovanni, Gastaldi Matteo, Liguori Rocco, Maestri Michelangelo, Pegoraro Elena, Polistena Barbara, Rodolico Carmelo, Habetswallner Francesco
Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), Sapienza University of Rome, Rome, Italy.
Neurol Ther. 2025 Aug 5. doi: 10.1007/s40120-025-00796-w.
Myasthenia gravis, a rare autoimmune disorder characterized by muscle weakness and fatigue, it is a mainly B-cell mediated condition with antibodies directed against the acetylcholine receptor or functionally related molecules at the neuromuscular junction. Corticosteroids are still the most used treatment, as they are cheap and characterized by a rapid response. However, their long-term administration is associated with frequent and often severe side effects.
We used the Expert Opinion methodology: a panel of eight neurologists, known to be experts in the management of MG patients, and one specialist in pharmacoeconomics, were brought together to discuss clinical relevant issues about the use of corticosteroids in MG.
Increasing doses of corticosteroids may temporarily exacerbate the symptoms of MG and clinical exacerbations can lead to severe consequences. In addition, prolonged chronic corticosteroid therapy carries a burden in terms of indirect costs due to side effects, which has prompted strategies to obtain the maximum benefits with minimal side effects.
The panel concludes that, in the near future, therapeutic strategies based on the use drugs with better tolerability and potentially lower direct and indirect costs, will be necessary.
重症肌无力是一种罕见的自身免疫性疾病，其特征为肌肉无力和疲劳，主要是由B细胞介导的病症，抗体针对神经肌肉接头处的乙酰胆碱受体或功能相关分子。皮质类固醇仍然是最常用的治疗方法，因为它们价格便宜且起效迅速。然而，长期使用会伴有频繁且往往严重的副作用。
我们采用专家意见法：召集了一组八名神经科医生（均为重症肌无力患者管理方面的知名专家）和一名药物经济学专家，共同讨论皮质类固醇在重症肌无力治疗中的临床相关问题。
皮质类固醇剂量增加可能会暂时加重重症肌无力的症状，而临床症状加重可能会导致严重后果。此外，长期慢性皮质类固醇治疗因副作用会带来间接成本负担，这促使人们寻求以最小副作用获得最大益处的策略。
该专家小组得出结论，在不久的将来，有必要采用基于使用耐受性更好且直接和间接成本可能更低的药物的治疗策略。
