Efgartigimod,a novel neonatal Fc receptor antagonist,has been approved for the treatment of adults with generalizedmyasthenia gravis who test positive for antibodies against the acetylcholine receptor(AChR).By promoti...Efgartigimod,a novel neonatal Fc receptor antagonist,has been approved for the treatment of adults with generalizedmyasthenia gravis who test positive for antibodies against the acetylcholine receptor(AChR).By promoting the degradation of pathogenic IgG antibodies,it effectively reduces the disease burden.This report presents two cases of herpes zoster occurring during efgartigimod therapy,raising concerns about its potential role in varicella-zoster virus(VZV)reactivation.Both patients had a history of thymoma and were receiving concurrent immunosuppressive therapy,which may have contributed to VZV reactivation.Notably,efgartigimod treatment was continued without further VZV recurrence over 10 and 6 months of follow-up,respectively.These cases highlight the importance of careful monitoring and consideration of VZV vaccination before initiating efgartigimod therapy in at-risk patients.Further research is needed to elucidate the relationship between Fc receptor inhibition and the risk of viral reactivation.展开更多
Myasthenia gravis is an acquired,humoral immunity-mediated autoimmune disease characterized by the production of autoantibodies that impair synaptic transmission at the neuromuscular junction.The intervention-mediated...Myasthenia gravis is an acquired,humoral immunity-mediated autoimmune disease characterized by the production of autoantibodies that impair synaptic transmission at the neuromuscular junction.The intervention-mediated clearance of immunoglobulin G(IgG)was shown to be effective in controlling the progression of the disease.The neonatal Fc receptor(FcRn)plays a key role in prolonging the serum half-life of IgG.Antagonizing FcRn to prevent its binding to IgG can accelerate the catabolism of the latter,resulting in decreased levels of IgG,including pathogenic autoantibodies,thereby achieving a therapeutic effect.In this review,we detail the substantial research progress,both basic and clinical,relating to the use of FcRn inhibitors in the treatment of myasthenia gravis.展开更多
目的对艾加莫德α治疗全身型重症肌无力的有效性、安全性和经济性进行快速卫生技术评估,为临床合理用药提供循证依据。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、CNKI、VIP、WanFang Data、SinoMed数据库及卫...目的对艾加莫德α治疗全身型重症肌无力的有效性、安全性和经济性进行快速卫生技术评估,为临床合理用药提供循证依据。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、CNKI、VIP、WanFang Data、SinoMed数据库及卫生技术评估机构官方网站,搜集艾加莫德α治疗全身型重症肌无力的卫生技术评估报告、系统评价/Meta分析和药物经济学研究,检索时限为建库至2024年6月21日。由2名研究者筛选文献、提取数据、评价文献质量,将研究结果汇总并进行定性描述分析。结果共纳入文献6篇,包括5篇系统评价/Meta分析和1篇药物经济学研究。有效性方面,与安慰剂相比,艾加莫德α可显著降低全身型重症肌无力患者的重症肌无力日常生活能力评分、重症肌无力定量评分和15项重症肌无力生活质量评分,差异有统计学意义(P<0.05),但与巴托利单抗、洛利昔珠单抗、依库珠单抗等其他生物制剂相比,各研究得出的结论不一致。安全性方面,艾加莫德α组与安慰剂组成年全身型重症肌无力患者的不良事件发生率差异无统计学意义(P>0.05),但与其他生物制剂相比,各研究得出的结论不一致。经济学方面,与传统疗法相比,艾加莫德α不具有成本-效果优势。结论艾加莫德α治疗全身型重症肌无力的疗效优于安慰剂,但与其他生物制剂相比无确切结论。在安全性方面,艾加莫德α在成年全身型重症肌无力患者中不良事件发生率与安慰剂相当,与其他生物制剂相比无确切结论。根据美国药物经济学数据,与传统疗法相比,艾加莫德α不具有经济学优势。展开更多
文摘Efgartigimod,a novel neonatal Fc receptor antagonist,has been approved for the treatment of adults with generalizedmyasthenia gravis who test positive for antibodies against the acetylcholine receptor(AChR).By promoting the degradation of pathogenic IgG antibodies,it effectively reduces the disease burden.This report presents two cases of herpes zoster occurring during efgartigimod therapy,raising concerns about its potential role in varicella-zoster virus(VZV)reactivation.Both patients had a history of thymoma and were receiving concurrent immunosuppressive therapy,which may have contributed to VZV reactivation.Notably,efgartigimod treatment was continued without further VZV recurrence over 10 and 6 months of follow-up,respectively.These cases highlight the importance of careful monitoring and consideration of VZV vaccination before initiating efgartigimod therapy in at-risk patients.Further research is needed to elucidate the relationship between Fc receptor inhibition and the risk of viral reactivation.
基金supported by the National Natural Science Foundation of China,No.U1604181the Joint Project of Medical Science and Technology Research Program of Henon Province,No.LHGJ20190078+1 种基金Henan Medical Education Research Project,No.Wjlx2020531Henan Province Key R&D and Promotion Special Project(Science and Technology Tackle),No.212102310834(all to JW)。
文摘Myasthenia gravis is an acquired,humoral immunity-mediated autoimmune disease characterized by the production of autoantibodies that impair synaptic transmission at the neuromuscular junction.The intervention-mediated clearance of immunoglobulin G(IgG)was shown to be effective in controlling the progression of the disease.The neonatal Fc receptor(FcRn)plays a key role in prolonging the serum half-life of IgG.Antagonizing FcRn to prevent its binding to IgG can accelerate the catabolism of the latter,resulting in decreased levels of IgG,including pathogenic autoantibodies,thereby achieving a therapeutic effect.In this review,we detail the substantial research progress,both basic and clinical,relating to the use of FcRn inhibitors in the treatment of myasthenia gravis.
文摘目的对艾加莫德α治疗全身型重症肌无力的有效性、安全性和经济性进行快速卫生技术评估,为临床合理用药提供循证依据。方法计算机检索PubMed、Embase、Web of Science、Cochrane Library、CNKI、VIP、WanFang Data、SinoMed数据库及卫生技术评估机构官方网站,搜集艾加莫德α治疗全身型重症肌无力的卫生技术评估报告、系统评价/Meta分析和药物经济学研究,检索时限为建库至2024年6月21日。由2名研究者筛选文献、提取数据、评价文献质量,将研究结果汇总并进行定性描述分析。结果共纳入文献6篇,包括5篇系统评价/Meta分析和1篇药物经济学研究。有效性方面,与安慰剂相比,艾加莫德α可显著降低全身型重症肌无力患者的重症肌无力日常生活能力评分、重症肌无力定量评分和15项重症肌无力生活质量评分,差异有统计学意义(P<0.05),但与巴托利单抗、洛利昔珠单抗、依库珠单抗等其他生物制剂相比,各研究得出的结论不一致。安全性方面,艾加莫德α组与安慰剂组成年全身型重症肌无力患者的不良事件发生率差异无统计学意义(P>0.05),但与其他生物制剂相比,各研究得出的结论不一致。经济学方面,与传统疗法相比,艾加莫德α不具有成本-效果优势。结论艾加莫德α治疗全身型重症肌无力的疗效优于安慰剂,但与其他生物制剂相比无确切结论。在安全性方面,艾加莫德α在成年全身型重症肌无力患者中不良事件发生率与安慰剂相当,与其他生物制剂相比无确切结论。根据美国药物经济学数据,与传统疗法相比,艾加莫德α不具有经济学优势。