美西汀治疗非营养不良性肌强直:六位医学专家的访谈

H. Boreham
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引用次数: 0

摘要

来自随机对照临床试验的证据表明,美西汀有效地控制肌强直症状,并具有良好的安全性。尽管如此,接受这种治疗的患者数量仍然有限,神经肌肉专家中心的医生可能没有完全意识到美西汀治疗的潜在益处,包括改善患者的生活质量(QoL),对日常生活活动的影响,以及良好的安全性,即使在年轻患者中也是如此。在“与专家会面”系列访谈中,五位在非营养不良性肌强直(NDM)治疗方面经验丰富的神经科医生和一位心脏病专家就支持美西汀治疗NDM的临床数据和现实证据提供了他们的专业见解。强调解决患者关切和鼓励治疗接受是优化美西汀治疗结果的关键步骤。医学专家强调,必须将美西汀有利的获益-风险概况,特别是在心脏安全问题和药物监测要求方面加以考虑。心脏病专家进一步解释说,根据他们的经验,与临床研究中使用的健康对照组相比,美西汀在NDM患者中的心脏安全性没有什么不同。在考虑NDM患者的抗肌强直治疗时,医学专家强调,决策应由肌强直的整体程度驱动。因此,医疗保健专业人员(HCP)需要超越基本的临床评估,以了解肌强直对患者日常生活的真正影响。总的来说,这些访谈强调了HCPs在利用临床数据和管理患者期望方面可以发挥的关键作用,以确保向NDM患者推荐美西汀时取得最大的治疗成功。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Mexiletine in the Treatment of Non-dystrophic Myotonia: Interviews with Six Medical Experts
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.
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