Mexiletine in the Treatment of Non-dystrophic Myotonia: Interviews with Six Medical Experts

Abstract

Evidence from randomised controlled clinical trials demonstrates that mexiletine effectively controls myotonic symptoms with a well-described safety profile. Despite this, there are still a limited number of patients on this treatment, and physicians from expert neuromuscular centres may not be fully aware of the potential benefits of mexiletine treatment, including improvements in patients’ quality of life (QoL), the impact on activities of daily living, and the favourable safety profile, even in younger patients. During this ‘Meet the Experts’ interview series, five neurologists experienced in the management of non-dystrophic myotonias (NDM), and one specialist cardiologist offered their expert insights on the clinical data and real-world evidence supporting the use of mexiletine in NDM. Addressing patient concerns and encouraging treatment acceptance were highlighted as key steps to optimise outcomes from mexiletine therapy. The medical experts emphasised the importance of contextualising the favourable benefit-to-risk profile of mexiletine, particularly regarding cardiac safety concerns and drug monitoring requirements. The expert cardiologist further explained that, in their experience, the cardiac safety profile of mexiletine was no different in patients with NDM when compared to healthy controls used in clinical studies when used as directed. When considering anti-myotonia treatment in patients with NDM, medical experts stressed that decision-making should be driven by the overall degree of myotonia. Healthcare professionals (HCP), therefore, need to look beyond basic clinical assessment to understand the true impact of myotonia on patients’ everyday lives. Overall, these interviews highlighted the critical role that HCPs can play in leveraging the clinical data and managing patient expectations to ensure maximum treatment success when recommending mexiletine to patients with NDM.