QUINIDINE SULFATE IN CONGENITAL SLOW CHANNEL MYASTHENIC SYNDROME

本研究的目的是确定硫酸奎尼丁是否会改善慢通道肌无力综合征患者的力量，并降低神经肌肉传递缺陷的严重程度。 肌肉力量的变化和神经肌肉传递的电生理测量将作为研究的终点。该议定书于1996年启动。 迄今为止，入组的2例患者在发生过敏反应后停用硫酸奎尼丁，过敏反应的特征为发热、肌痛，1例患者的肝转氨酶一过性升高。 停药后，所有症状均消退。未报告其他不良反应。 对奎尼丁发生过敏反应的两名患者均接受了氟西汀治疗，临床反应良好，无不良反应。 在一次国家会议和一次国际会议上介绍了前6例患者长达18个月随访的数据。 两份描述这项工作的手稿已经出版（《纽约科学院年鉴》和《神经病学年鉴》）。 迄今为止，所有入组的患者在力量和神经肌肉传递功能的临床和电生理测量方面均显示出显著的客观改善。

The aim of the study is to determine if quinidine sulfate will improve strength and reduce the severity of the neuromuscular transmission defects in patients with slow channel myasthenic syndrome. Changes in muscle strength and electrophysiological measures of neuromuscular transmission performed will serve as end points for the study. The protocol was initiated in 1996. Two of the patients enrolled to date discontinued quinidine sulfate after developing an allergic reaction characterized by fever, myalgias, and in one case transient elevation in liver transaminases. All symptoms resolved when the drug was discontinued. No other adverse effects were reported. Both of the patients who developed an allergic reaction to quinidine have been placed on Fluoxetine with a good clinical response and no adverse effects. The data from the first 6 patients with up to 18 months follow up has been presented at one national and one international meeting. Two manuscripts describing the work have been published (Annals New York Academy of Sciences, and Annals of Neurology). All patients enrolled to date have shown significant objective improvement in clinical and electrophysiological measures of strength and neuromuscular transmission function.