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Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion.

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Title: Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion.
Authors: Mantegazza, Renato (AUTHOR), Saccà, Francesco (AUTHOR), Antonini, Giovanni (AUTHOR), Bonifati, Domenico Marco (AUTHOR), Evoli, Amelia (AUTHOR), Habetswallner, Francesco (AUTHOR), Liguori, Rocco (AUTHOR), Pegoraro, Elena (AUTHOR), Rodolico, Carmelo (AUTHOR), Schenone, Angelo (AUTHOR), Sgarzi, Manlio (AUTHOR), Pappagallo, Giovanni (AUTHOR)
Source: Neurological Sciences. Dec2024, Vol. 45 Issue 12, p5671-5683. 13p.
Subjects: Cholinergic receptors, Therapeutics, Protein-tyrosine kinases, Immunosuppressive agents, Complement activation, Eculizumab, Myasthenia gravis, Off-label use (Drugs)
Abstract: Myasthenia gravis (MG) is a rare, autoimmune, neurological disorder. Most MG patients have autoantibodies against acetylcholine receptors (AChRs). Some have autoantibodies against muscle-specific tyrosine kinase (MuSK) or lipoprotein-receptor-related protein 4 (LRP4), and some are seronegative. Standard of care, which includes anti-cholinesterase drugs, thymectomy, corticosteroids (CS), and off-label use of non-steroidal immunosuppressive drugs (NSISTs), is bounded by potential side effects and limited efficacy in refractory generalized MG (gMG) patients. This highlights the need for new therapeutic approaches for MG. Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG. A panel of 11 experts met to discuss unmet therapeutic needs in the acute and chronic phases of the disease, as well as the standard of care for refractory patients. Survival was emphasized as an acute phase outcome. In the chronic phase, persistent remission and early recognition of exacerbations to prevent myasthenic crisis and respiratory failure were considered crucial. Refractory patients require treatments with fast onset of action, improved tolerability, and the ability to slow disease progression and increase life expectancy. The Panel agreed that eculizumab would presumably meet the therapeutic needs of many refractory gMG patients. The panel concluded that the unmet needs of current standard of care treatments for gMG are significant. Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs. [ABSTRACT FROM AUTHOR]
Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
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  Data: <searchLink fieldCode="AR" term="%22Mantegazza%2C+Renato%22">Mantegazza, Renato</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Saccà%2C+Francesco%22">Saccà, Francesco</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Antonini%2C+Giovanni%22">Antonini, Giovanni</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Bonifati%2C+Domenico+Marco%22">Bonifati, Domenico Marco</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Evoli%2C+Amelia%22">Evoli, Amelia</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Habetswallner%2C+Francesco%22">Habetswallner, Francesco</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Liguori%2C+Rocco%22">Liguori, Rocco</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Pegoraro%2C+Elena%22">Pegoraro, Elena</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Rodolico%2C+Carmelo%22">Rodolico, Carmelo</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Schenone%2C+Angelo%22">Schenone, Angelo</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Sgarzi%2C+Manlio%22">Sgarzi, Manlio</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Pappagallo%2C+Giovanni%22">Pappagallo, Giovanni</searchLink> (AUTHOR)
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  Data: Myasthenia gravis (MG) is a rare, autoimmune, neurological disorder. Most MG patients have autoantibodies against acetylcholine receptors (AChRs). Some have autoantibodies against muscle-specific tyrosine kinase (MuSK) or lipoprotein-receptor-related protein 4 (LRP4), and some are seronegative. Standard of care, which includes anti-cholinesterase drugs, thymectomy, corticosteroids (CS), and off-label use of non-steroidal immunosuppressive drugs (NSISTs), is bounded by potential side effects and limited efficacy in refractory generalized MG (gMG) patients. This highlights the need for new therapeutic approaches for MG. Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG. A panel of 11 experts met to discuss unmet therapeutic needs in the acute and chronic phases of the disease, as well as the standard of care for refractory patients. Survival was emphasized as an acute phase outcome. In the chronic phase, persistent remission and early recognition of exacerbations to prevent myasthenic crisis and respiratory failure were considered crucial. Refractory patients require treatments with fast onset of action, improved tolerability, and the ability to slow disease progression and increase life expectancy. The Panel agreed that eculizumab would presumably meet the therapeutic needs of many refractory gMG patients. The panel concluded that the unmet needs of current standard of care treatments for gMG are significant. Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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