Ravulizumab and Efgartigimod in Myasthenia Gravis

被引:6
作者
Stascheit, Frauke [1 ,2 ,3 ,4 ]
Sousa, Carla Daiane Ferreira de [2 ,3 ,5 ]
Aigner, Annette [2 ,3 ,6 ,7 ]
Behrens, Malina [2 ,3 ,5 ]
Keller, Christian W. [2 ,3 ,5 ]
Klotz, Luisa [2 ,3 ,5 ]
Lehnerer, Sophie [1 ,2 ,3 ,4 ]
Stein, Maike [1 ,2 ,3 ,4 ,7 ]
Herdick, Meret [1 ,2 ,3 ,4 ]
Doksani, Paolo [1 ,2 ,3 ,4 ]
Gerischer, Lea M. [1 ,2 ,3 ,4 ]
Hoffmann, Sarah [1 ,2 ,3 ,4 ]
Lazaridis, Konstantinos [2 ,3 ,8 ]
Tzartos, John [2 ,3 ,9 ]
Wiendl, Heinz [2 ,3 ,5 ]
Meisel, Andreas [1 ,2 ,3 ,4 ,7 ]
Luenemann, Jan D. [5 ]
机构
[1] Charite Univ Med Berlin, Dept Neurol Expt Neurol, Berlin, Germany
[2] Free Univ Berlin, Berlin, Germany
[3] Humboldt Univ, Berlin, Germany
[4] Charite Univ Med Berlin, Neurosci Clin Res Ctr, Berlin, Germany
[5] Univ Hosp Munster, Inst Translat Neurol, Dept Neurol, Munster, Germany
[6] Charite Univ Med Berlin, Inst Biometry & Clin Epidemiol, Berlin, Germany
[7] Charite Univ Med Berlin, Ctr Stroke Res Berlin, Berlin, Germany
[8] Hellenic Pasteur Inst, Dept Immunol, Athens, Greece
[9] Natl & Kapodistrian Univ Athens, Attikon Univ Hosp, Sch Med, Neurol Dept 2, Athens, Greece
关键词
D O I
10.1212/NXI.0000000000200331
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Background and ObjectivesBiologics that target pathogenic antibodies (Abs) and their effector functions such as the complement inhibitor ravulizumab and the neonatal Fc receptor agonist efgartigimod have recently been approved for the treatment of acetylcholine receptor (AChR)-Ab-positive myasthenia gravis (MG), but comparative studies are lacking.MethodsIn a prospective, exploratory real-world study, we assessed clinical efficacy, safety, and biological effects of ravulizumab and efgartigimod treatment initiation. Myasthenia Gravis-Activities of Daily Living and Quantitative Myasthenia Gravis scores were used as clinical endpoints. Ab effector functions were determined by AChR-Ab-dependent complement activation and phagocytosis assays and systemic complement activation profiling.ResultsWe observed similar moderate short-term efficacy of ravulizumab and efgartigimod in achieving clinical improvement. Ravulizumab reduced systemic terminal complement activation, but neither treatment showed significant effects on complement pathways proximal to C5 or functional capacities of AChR-Abs. Both treatment modalities were well tolerated with no serious adverse events reported.DiscussionClinical benefits obtained with ravulizumab and efgartigimod can be remarkably heterogeneous in daily clinical practice. Neither treatment relevantly changed effector functions of pathogenic AChR-Abs, supporting the concept that durable disease control in MG requires continuous administration of both fast-acting agents.Classification of EvidenceThis study provides Class III evidence that in AChR-Ab-positive patients with generalized MG, ravulizumab and efgartigimod provide comparable modest improvement in MG functional scales.
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页数:9
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