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Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis

Myasthenia gravis (MG) is an autoimmune disease primarily mediated by acetylcholine receptor antibodies (AChR-Ab), cellular immune dependence, and complement system involvement. Since the AChR on the postsynaptic membrane is destroyed by an immune attack, sufficient endplate potential cannot be gene...

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Autores principales: Xiao, Hai, Wu, Ka, Liang, Xiaoliu, Li, Rong, Lai, Keng Po
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8384962/
https://www.ncbi.nlm.nih.gov/pubmed/34456922
http://dx.doi.org/10.3389/fimmu.2021.715036
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author Xiao, Hai
Wu, Ka
Liang, Xiaoliu
Li, Rong
Lai, Keng Po
author_facet Xiao, Hai
Wu, Ka
Liang, Xiaoliu
Li, Rong
Lai, Keng Po
author_sort Xiao, Hai
collection PubMed
description Myasthenia gravis (MG) is an autoimmune disease primarily mediated by acetylcholine receptor antibodies (AChR-Ab), cellular immune dependence, and complement system involvement. Since the AChR on the postsynaptic membrane is destroyed by an immune attack, sufficient endplate potential cannot be generated, resulting in the development of a synaptic transmission disorder at the neuromuscular junction and in muscle weakness. The role of the complement system in MG has been demonstrated in animal models and clinical tests, and it has been determined that complement inhibition in patients with MG can prevent disease induction and reverse its progression. Eculizumab is a humanized monoclonal antibody that inhibits the cleavage of complement protein C5 and prevents autoimmune damage; additionally, it has received subsequent approval by the Federal Drug Administration of the United States for MG treatment. However, various concerns regarding the use of eculizumab persist. In this review, we have discussed the treatment time, cost effectiveness, long-term efficacy, and tolerability of eculizumab for MG treatment. We have also summarized historical information and have presented perspectives on this new therapeutic modality.
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spelling pubmed-83849622021-08-26 Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis Xiao, Hai Wu, Ka Liang, Xiaoliu Li, Rong Lai, Keng Po Front Immunol Immunology Myasthenia gravis (MG) is an autoimmune disease primarily mediated by acetylcholine receptor antibodies (AChR-Ab), cellular immune dependence, and complement system involvement. Since the AChR on the postsynaptic membrane is destroyed by an immune attack, sufficient endplate potential cannot be generated, resulting in the development of a synaptic transmission disorder at the neuromuscular junction and in muscle weakness. The role of the complement system in MG has been demonstrated in animal models and clinical tests, and it has been determined that complement inhibition in patients with MG can prevent disease induction and reverse its progression. Eculizumab is a humanized monoclonal antibody that inhibits the cleavage of complement protein C5 and prevents autoimmune damage; additionally, it has received subsequent approval by the Federal Drug Administration of the United States for MG treatment. However, various concerns regarding the use of eculizumab persist. In this review, we have discussed the treatment time, cost effectiveness, long-term efficacy, and tolerability of eculizumab for MG treatment. We have also summarized historical information and have presented perspectives on this new therapeutic modality. Frontiers Media S.A. 2021-08-11 /pmc/articles/PMC8384962/ /pubmed/34456922 http://dx.doi.org/10.3389/fimmu.2021.715036 Text en Copyright © 2021 Xiao, Wu, Liang, Li and Lai https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Immunology
Xiao, Hai
Wu, Ka
Liang, Xiaoliu
Li, Rong
Lai, Keng Po
Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title_full Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title_fullStr Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title_full_unstemmed Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title_short Clinical Efficacy and Safety of Eculizumab for Treating Myasthenia Gravis
title_sort clinical efficacy and safety of eculizumab for treating myasthenia gravis
topic Immunology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8384962/
https://www.ncbi.nlm.nih.gov/pubmed/34456922
http://dx.doi.org/10.3389/fimmu.2021.715036
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