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Understanding the burden of refractory myasthenia gravis
Myasthenia gravis (MG) is an autoantibody-mediated disease that compromises the acetylcholine receptors or associated structures of the postsynaptic membrane of the neuromuscular junction. This leads to impaired neuromuscular transmission and subsequent fluctuating fatigability and weakness of ocula...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6399761/ https://www.ncbi.nlm.nih.gov/pubmed/30854027 http://dx.doi.org/10.1177/1756286419832242 |
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author | Schneider-Gold, Christiane Hagenacker, Tim Melzer, Nico Ruck, Tobias |
author_facet | Schneider-Gold, Christiane Hagenacker, Tim Melzer, Nico Ruck, Tobias |
author_sort | Schneider-Gold, Christiane |
collection | PubMed |
description | Myasthenia gravis (MG) is an autoantibody-mediated disease that compromises the acetylcholine receptors or associated structures of the postsynaptic membrane of the neuromuscular junction. This leads to impaired neuromuscular transmission and subsequent fluctuating fatigability and weakness of ocular, bulbar, and limb skeletal muscles. Over the past few decades, there have been significant advances in our understanding of the disease pathophysiology and improvements in prognosis due to intensive care medicine and immunomodulation. Despite this, an estimated 10–20% of patients with MG do not achieve an adequate response, are intolerant to conventional treatment, or require chronic treatment with intravenous immunoglobulins or plasma separation procedures. Such patients are regarded as having MG that is ‘refractory’ to treatment and may represent a distinct clinical subgroup. Because the majority of patients with MG have well-controlled disease, the burden of illness in the minority with refractory disease is poorly understood and may be underestimated. However, clinically these patients are liable to experience extreme fatigue, considerable disability owing to uncontrolled symptoms, and frequent myasthenic crises and hospitalizations. Both acute adverse effects and an increased risk of comorbidity from treatment regimens may contribute to reduced quality of life. As yet, little is known concerning the impact of refractory MG on mental health and health-related quality of life. This review aims to highlight the burden of disease and unmet needs in patients with refractory MG. |
format | Online Article Text |
id | pubmed-6399761 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | SAGE Publications |
record_format | MEDLINE/PubMed |
spelling | pubmed-63997612019-03-08 Understanding the burden of refractory myasthenia gravis Schneider-Gold, Christiane Hagenacker, Tim Melzer, Nico Ruck, Tobias Ther Adv Neurol Disord Review Myasthenia gravis (MG) is an autoantibody-mediated disease that compromises the acetylcholine receptors or associated structures of the postsynaptic membrane of the neuromuscular junction. This leads to impaired neuromuscular transmission and subsequent fluctuating fatigability and weakness of ocular, bulbar, and limb skeletal muscles. Over the past few decades, there have been significant advances in our understanding of the disease pathophysiology and improvements in prognosis due to intensive care medicine and immunomodulation. Despite this, an estimated 10–20% of patients with MG do not achieve an adequate response, are intolerant to conventional treatment, or require chronic treatment with intravenous immunoglobulins or plasma separation procedures. Such patients are regarded as having MG that is ‘refractory’ to treatment and may represent a distinct clinical subgroup. Because the majority of patients with MG have well-controlled disease, the burden of illness in the minority with refractory disease is poorly understood and may be underestimated. However, clinically these patients are liable to experience extreme fatigue, considerable disability owing to uncontrolled symptoms, and frequent myasthenic crises and hospitalizations. Both acute adverse effects and an increased risk of comorbidity from treatment regimens may contribute to reduced quality of life. As yet, little is known concerning the impact of refractory MG on mental health and health-related quality of life. This review aims to highlight the burden of disease and unmet needs in patients with refractory MG. SAGE Publications 2019-03-01 /pmc/articles/PMC6399761/ /pubmed/30854027 http://dx.doi.org/10.1177/1756286419832242 Text en © The Author(s), 2019 http://www.creativecommons.org/licenses/by-nc/4.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (http://www.creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
spellingShingle | Review Schneider-Gold, Christiane Hagenacker, Tim Melzer, Nico Ruck, Tobias Understanding the burden of refractory myasthenia gravis |
title | Understanding the burden of refractory myasthenia gravis |
title_full | Understanding the burden of refractory myasthenia gravis |
title_fullStr | Understanding the burden of refractory myasthenia gravis |
title_full_unstemmed | Understanding the burden of refractory myasthenia gravis |
title_short | Understanding the burden of refractory myasthenia gravis |
title_sort | understanding the burden of refractory myasthenia gravis |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6399761/ https://www.ncbi.nlm.nih.gov/pubmed/30854027 http://dx.doi.org/10.1177/1756286419832242 |
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