QUINIDINE SULFATE IN CONGENITAL SLOW CHANNEL MYASTHENIC SYNDROME

这项研究的目的是确定硫酸奎尼丁是否能提高慢通道肌无力综合征患者的强度并减轻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。到目前为止登记的两名患者在出现以发热、肌痛为特征的过敏反应后停用硫酸奎尼丁，其中一例肝脏转氨酶一过性升高。当药物停止使用时，所有症状都消失了。没有其他不良反应的报道。这两名对奎尼丁产生过敏反应的患者都接受了氟西汀治疗，临床反应良好，没有不良反应。前6名患者长达18个月的随访数据已在一次全国会议和一次国际会议上公布。描述这项工作的两份手稿已经出版(《纽约科学院年鉴》和《神经学年鉴》)。到目前为止，所有登记的患者在临床和电生理测量力量和神经肌肉传递功能方面都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.