笪宇威教授谈重症肌无力治疗进展:从困境突破到精准管理新路径
Xin Lang Cai Jing·2025-12-21 11:28

Core Viewpoint - The article discusses the treatment challenges and advancements in therapies for generalized myasthenia gravis (gMG), emphasizing the need for targeted therapies to address the limitations of traditional treatments [1][2][8]. Treatment Challenges - Traditional non-specific immunotherapy, including corticosteroids and non-steroidal immunosuppressants, has significant limitations, including adverse effects from long-term corticosteroid use and slow onset of action for other immunosuppressants [2][3]. - Approximately 10% to 20% of patients show limited response to conventional treatments, leading to a subset of patients developing "refractory gMG" [2][3]. - The heterogeneity of MG results in varied patient responses to medications, complicating the selection of effective treatment plans [2][3]. Advancements in Targeted Therapies - Targeted therapies for gMG are categorized into several classes, including FcRn antagonists, B-cell targeted therapies, and complement inhibitors [3][4]. - FcRn antagonists, such as efgartigimod, accelerate the degradation of pathogenic antibodies, reducing their levels [4]. - B-cell targeted therapies include monoclonal antibodies like rituximab and inebilizumab, which have shown efficacy in improving clinical symptoms and reducing corticosteroid dosage [4][6]. - Complement inhibitors, such as eculizumab and ravulizumab, block the complement pathway, preventing immune damage to neuromuscular junctions [4][6]. Clinical Evidence and Guidelines - Clinical studies indicate that treatments like eculizumab significantly improve MG-ADL and QMG scores within weeks, with a notable percentage of patients able to reduce corticosteroid use [5][6]. - The 2025 guidelines emphasize achieving "minimal symptom expression" as a treatment goal, highlighting the importance of long-term maintenance therapy [3][8]. - The CHAMPION MG OLE study demonstrated a 71.3% reduction in clinical deterioration events after 60 weeks of treatment with eculizumab [5][6]. Future Directions - The article advocates for a shift from traditional stepwise treatment to early targeted interventions and personalized treatment strategies for gMG patients [7][8]. - Emphasis is placed on the need for early intervention in high-activity gMG patients to prevent irreversible damage and improve quality of life [7][8]. - A comprehensive management model that includes rapid control of symptoms, long-term maintenance, and safe tapering of corticosteroids is essential for effective gMG treatment [7][8].