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Recommendations of an expert group for the cardiac assessment of non-dystrophic myotonia adult patients treated with mexiletine.

Neuromuscul Disord

November 2024

Cardiology Department, CHU de Caen, UR 4650 Unicaen, Avenue de la Côte de Nacre CS 30001, 14033 Caen France.

Mexiletine (NaMuscla™) is indicated for the symptomatic treatment of myotonia in adults with non-dystrophic myotonia. A cardiac assessment is required as mexiletine may have a pro-arrhythmic effect. Long-term safety data supporting the use of mexiletine in patients with non-dystrophic myotonia combined with the extensive clinical experience of an expert group resulted in creation of an algorithm for cardiac monitoring of patients treated with mexiletine.

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Development, validation and long-term evaluation of a liquid chromatography-tandem mass spectrometry method for simultaneous quantification of amiodarone, desethylamiodarone and mexiletine in human plasma and serum.

J Chromatogr B Analyt Technol Biomed Life Sci

August 2024

Department of Pharmacy and Clinical Pharmacology, University of Amsterdam, Amsterdam UMC Location AMC, Amsterdam, The Netherlands; Medicines for Society Platform, Amsterdam UMC - University of Amsterdam, Amsterdam, The Netherlands.

Amiodarone and mexiletine are used for ventricular arrhythmias, for which a combination therapy of both anti-arrhythmic drugs (AADs) is not uncommon. Therapeutic drug monitoring (TDM) can be beneficial for clinical guidance of therapy, especially to correctly identify adverse events. Desethylamiodarone, the active metabolite of amiodarone, accumulates over time and is associated with serious adverse events.

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Introduction: The Individualized Neuromuscular Quality of Life Questionnaire (INQoL) is used to measure quality of life in neuromuscular disorders such as non-dystrophic myotonia (NDM). Here we report methods to estimate utilities, with a focus on NDM, from this questionnaire based on two preference elicitation exercises.

Methods: Eight items from the INQoL were selected with input from three neuromuscular disorder clinical experts with expertise in treating NDM.

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Background: The rare nature of dystrophic and non-dystrophic myotonia has limited the available evidence on the efficacy of mexiletine as a potential treatment. To address this gap, we conducted a systematic review and meta-analysis to evaluate the effectiveness and safety of mexiletine for both dystrophic and non-dystrophic myotonic patients.

Methods: The search was conducted on various electronic databases up to March 2023, for randomized clinical trials (RCTs) comparing mexiletine versus placebo in myotonic patients.

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