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

IF 4.3 3区 材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC
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.

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
CiteScore
7.20
自引率
4.30%
发文量
567
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