Expert Insights from a Delphi-driven Neurologists' Panel: Real-world Mexiletine use in Patients with Myotonic Disorders in Italy.

IF 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2024-01-01 DOI:10.3233/JND-230115

Dario Lidonnici, Pietro Brambilla, Roberto Ravasio, Alla Zozulya-Weidenfeller, Annette Beiderbeck, Mariska van Aswegen, Rosa Oliveira, Valeria A Sansone

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Abstract

Background: Myotonic disorders, such as non-dystrophic myotonias (NDMs) and myotonic dystrophies (DMs) are characterized by a delay in muscle relaxation after a contraction stimulus. There is general consensus that protocols to treat myotonia need to be implemented.

Objective: Mexiletine is the only pharmacological agent approved for the symptomatic treatment of myotonia in adult patients with NDM and is considered to be the first-line treatment for DMs; however, its production in Italy was halted in 2022 making its availability to patients problematic.

Methods: A panel of 8 Italian neurologists took part in a two-round Delphi panel between June and October 2022, analyzing the current use of mexiletine in Italian clinical practice.

Results: The panelists assist 1126 patients (69% DM type1, 18% NDM and 13% DM type2). Adult NDM patients receive, on average, 400-600 mg of mexiletine hydrochloride (HCl) while adult DM patients receive 100-600 mg, per day in the long-term. The severity of symptoms is considered the main reason to start mexiletine treatment for both NDM and DM patients. Mexiletine is reckoned to have a clinical impact for both NDM and DM patients, but currently drug access is problematic.

Conclusions: Mexiletine treatment is recognized to have a role in the reduction of the symptomatic burden for NDM and DM patients. Patient management could be improved by facilitating access to therapy and developing new drug formulations.

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来自德尔菲神经科专家小组的专家见解：意大利肌张力障碍患者使用 Mexiletine 的实际情况。

背景：肌张力障碍，如非萎缩性肌张力障碍（NDMs）和肌张力营养不良症（DMs），其特征是肌肉在受到收缩刺激后迟迟不能松弛。人们普遍认为需要实施治疗肌张力障碍的方案：然而，该药物在意大利的生产已于 2022 年停止，因此患者难以获得该药物：由 8 位意大利神经科专家组成的小组在 2022 年 6 月至 10 月期间参加了两轮德尔菲小组讨论，分析了目前在意大利临床实践中使用美西律汀的情况：小组成员共协助了 1126 名患者（69% 为 DM1 型，18% 为 NDM 型，13% 为 DM2 型）。成年 NDM 患者平均每天服用 400-600 毫克盐酸美西律汀（HCl），而成年 DM 患者每天长期服用 100-600 毫克。症状的严重程度被认为是 NDM 和 DM 患者开始接受美西雷定治疗的主要原因。据估计，美西雷定对NDM和DM患者都有临床疗效，但目前药物获取存在问题：美西列汀治疗被认为可以减轻 NDM 和 DM 患者的症状负担。通过促进治疗的可及性和开发新的药物制剂，可以改善对患者的管理。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.