Department of Neurology, Medical Faculty, Heinrich-Heine University, Düsseldorf, Germany.
Servei de Neurologia-Neuroimmunologia, Centre d'Esclerosi Múltiple de Catalunya (Cemcat), Hospital Universitario Vall d'Hebron, Barcelona, Spain.
CNS Drugs. 2018 Jul;32(7):637-651. doi: 10.1007/s40263-018-0536-2.
The oral potassium channel blocker 4-aminopyridine has been used in various neurological conditions for decades. Numerous case reports and studies have supported its clinical efficacy in ameliorating the clinical presentation of certain neurological disorders. However, its short half-life, erratic drug levels, and safety-related dose restrictions limited its use as a self-compounded drug in clinical practice. This changed with the introduction of a prolonged-release formulation, which was successfully tested in patients with multiple sclerosis. It was fully approved by the US FDA in January 2010 but initially received only conditional approval from the European Medicines Agency (EMA) in July 2011. After additional clinical studies, this conditional approval was changed to unrestricted approval in August 2017. This article reviews and discusses these recent studies and places aminopyridines and their clinical utility into the context of a broader spectrum of neurological disorders, where clinical efficacy has been suggested. In 2010, prolonged-release 4-aminopyridine became the first drug specifically licensed to improve walking in patients with multiple sclerosis. About one-third of patients across disease courses benefit from this treatment. In addition, various reports indicate clinical efficacy beyond multiple sclerosis, which may broaden its use in clinical practice.
口服钾通道阻滞剂 4-氨基吡啶已在各种神经疾病中使用了数十年。大量的病例报告和研究支持其在改善某些神经疾病临床表现方面的临床疗效。然而，其半衰期短、药物水平不稳定以及与安全性相关的剂量限制限制了其在临床实践中作为自配药物的使用。这种情况随着长效制剂的引入而改变，该制剂已在多发性硬化症患者中成功进行了测试。它于 2010 年 1 月被美国 FDA 完全批准，但最初仅在 2011 年 7 月获得欧洲药品管理局（EMA）的有条件批准。经过额外的临床研究，这种有条件的批准在 2017 年 8 月改为无限制批准。本文回顾和讨论了这些最近的研究，并将氨基吡啶及其临床用途置于更广泛的神经疾病范围内进行讨论，在这些疾病中已经显示出了临床疗效。2010 年，长效 4-氨基吡啶成为第一种专门获准改善多发性硬化症患者行走能力的药物。大约三分之一的患者在疾病过程中受益于这种治疗。此外，各种报告表明其在多发性硬化症之外的临床疗效，这可能会扩大其在临床实践中的应用。
