Refocusing generalized myasthenia gravis: Patient burden, disease profiles, and the role of evolving therapy
Background and purpose Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a val...
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| Vydané v: | European journal of neurology Ročník 31; číslo 6; s. e16180 - n/a |
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| Hlavní autori: | , , , , |
| Médium: | Journal Article |
| Jazyk: | English |
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England
John Wiley & Sons, Inc
01.06.2024
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| ISSN: | 1351-5101, 1468-1331, 1468-1331 |
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| Abstract | Background and purpose
Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG.
Methods
Narrative review was made of publications identified via searches of PubMed and selected congresses (January 2000–September 2022).
Results
New consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision‐making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management.
Conclusions
gMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease. |
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| AbstractList | Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG.BACKGROUND AND PURPOSEGeneralized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG.Narrative review was made of publications identified via searches of PubMed and selected congresses (January 2000-September 2022).METHODSNarrative review was made of publications identified via searches of PubMed and selected congresses (January 2000-September 2022).New consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision-making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management.RESULTSNew consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision-making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management.gMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease.CONCLUSIONSgMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease. Background and purposeGeneralized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG.MethodsNarrative review was made of publications identified via searches of PubMed and selected congresses (January 2000–September 2022).ResultsNew consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision‐making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management.ConclusionsgMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease. Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG. Narrative review was made of publications identified via searches of PubMed and selected congresses (January 2000-September 2022). New consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision-making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management. gMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease. Background and purpose Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent disease fluctuations, and varying response to therapy. The recent availability of new pharmacologic therapies presents a valuable opportunity to reevaluate how this disease is classified, assessed, and managed and identify new ways to improve the clinical care of patients with gMG. Methods Narrative review was made of publications identified via searches of PubMed and selected congresses (January 2000–September 2022). Results New consensus definitions are required to ensure consistency, to better characterize patients, and to identify patients who will benefit from specific drugs and earlier use of these agents. There is a need for more frequent, standardized patient assessment to identify the cause of motor function deficits, provide a clearer picture of the disease burden and its impact on daily living and quality of life (QoL), and better support treatment decision‐making. Novel approaches that target different components of the immune system will play a role in more precise treatment of patients with gMG, alongside the development of new algorithms to guide individualized patient management. Conclusions gMG has a physical, mental, and social impact, resulting in a considerable burden of disease and substantially decreased QoL, despite standard treatments. The availability of novel, targeted treatments that influence key pathological mediators of gMG, together with new biomarkers, offers the potential to optimize patient management and ultimately enables a greater number of patients to achieve minimal manifestation status and a reduced burden of disease. |
| Author | Saccà, Francesco Schneider‐Gold, Christiane Cortés‐Vicente, Elena Salort‐Campana, Emmanuelle Jacob, Saiju |
| Author_xml | – sequence: 1 givenname: Francesco orcidid: 0000-0002-1323-6317 surname: Saccà fullname: Saccà, Francesco organization: Federico II University – sequence: 2 givenname: Emmanuelle surname: Salort‐Campana fullname: Salort‐Campana, Emmanuelle organization: Assistance Publique–Hopitaux de Marseille – sequence: 3 givenname: Saiju surname: Jacob fullname: Jacob, Saiju organization: University of Birmingham – sequence: 4 givenname: Elena orcidid: 0000-0002-1428-1072 surname: Cortés‐Vicente fullname: Cortés‐Vicente, Elena organization: Instituto de Salud Carlos III – sequence: 5 givenname: Christiane orcidid: 0000-0002-9232-201X surname: Schneider‐Gold fullname: Schneider‐Gold, Christiane email: christiane.schneider-gold@ruhr-uni-bochum.de organization: St. Josef Hospital of Ruhr‐University Bochum |
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Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease... Generalized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease course, frequent... Background and purposeGeneralized myasthenia gravis (gMG) continues to present significant challenges for clinical management due to an unpredictable disease... |
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| SubjectTerms | Algorithms Availability Biomarkers classification Cost of Illness Decision making disease burden generalized myasthenia gravis Health services Human motion Humans Immune system Immunosuppressive agents Myasthenia gravis Myasthenia Gravis - drug therapy Myasthenia Gravis - therapy Neuromuscular junctions pathophysiology Patients Quality of Life Social impact targeted therapy |
| Title | Refocusing generalized myasthenia gravis: Patient burden, disease profiles, and the role of evolving therapy |
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