Long-term Safety and Efficacy of Mexiletine in Myotonic Dystrophy Types 1 and 2
© 2021 American Academy of Neurology..
BACKGROUND AND OBJECTIVE: Myotonic dystrophy types 1 and 2 are progressive multisystem genetic disorders whose core clinical feature is myotonia. Mexiletine, an antagonist of voltage-gated sodium channels, is a recommended antimyotonic agent in the nondystrophic myotonias, but its use in myotonic dystrophy is limited because of lack of data regarding its long-term efficacy and safety profile.
METHODS: To address this issue, this study retrospectively evaluated patients with myotonic dystrophy receiving mexiletine over a mean time period of 32.9 months (range 0.1-216 months).
RESULTS: This study demonstrated that 96% of patients reported some improvement in myotonia symptoms with mexiletine treatment. No clinically relevant cardiac adverse events were associated with the long-term use of mexiletine.
CONCLUSIONS: These findings support that mexiletine is both safe and effective when used long-term in myotonic dystrophy.
CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that mexiletine is a well-tolerated and effective treatment for myotonic dystrophy types 1 and 2.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:11 |
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| Enthalten in: |
Neurology. Clinical practice - 11(2021), 5 vom: 22. Okt., Seite e682-e685 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Mousele, Christina [VerfasserIn] |
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| Anmerkungen: |
Date Revised 20.03.2024 published: Print Citation Status PubMed-not-MEDLINE |
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| doi: |
10.1212/CPJ.0000000000001073 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM333779770 |
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| 500 | |a Date Revised 20.03.2024 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status PubMed-not-MEDLINE | ||
| 520 | |a © 2021 American Academy of Neurology. | ||
| 520 | |a BACKGROUND AND OBJECTIVE: Myotonic dystrophy types 1 and 2 are progressive multisystem genetic disorders whose core clinical feature is myotonia. Mexiletine, an antagonist of voltage-gated sodium channels, is a recommended antimyotonic agent in the nondystrophic myotonias, but its use in myotonic dystrophy is limited because of lack of data regarding its long-term efficacy and safety profile | ||
| 520 | |a METHODS: To address this issue, this study retrospectively evaluated patients with myotonic dystrophy receiving mexiletine over a mean time period of 32.9 months (range 0.1-216 months) | ||
| 520 | |a RESULTS: This study demonstrated that 96% of patients reported some improvement in myotonia symptoms with mexiletine treatment. No clinically relevant cardiac adverse events were associated with the long-term use of mexiletine | ||
| 520 | |a CONCLUSIONS: These findings support that mexiletine is both safe and effective when used long-term in myotonic dystrophy | ||
| 520 | |a CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that mexiletine is a well-tolerated and effective treatment for myotonic dystrophy types 1 and 2 | ||
| 650 | 4 | |a Journal Article | |
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| 700 | 1 | |a Pitceathly, Robert D S |e verfasserin |4 aut | |
| 700 | 1 | |a Hanna, Michael G |e verfasserin |4 aut | |
| 700 | 1 | |a MacDonald, Susan |e verfasserin |4 aut | |
| 700 | 1 | |a Savvatis, Konstantinos |e verfasserin |4 aut | |
| 700 | 1 | |a Carr, Aisling |e verfasserin |4 aut | |
| 700 | 1 | |a Turner, Christopher |e verfasserin |4 aut | |
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