Abstract：
The aim of this study was to establish whether bumetanide can abort an acute attack of weakness in patients with HypoPP. This was a randomised, double-blind, cross-over, placebo-controlled phase II clinical trial. Focal attack of weakness was induced by isometric exercise of ADM followed by rest (McManis protocol). Participants had two study visits and received either placebo or 2 mg bumetanide at attack onset (defined as 40 % decrement in the abductor digiti minimi CMAP amplitude from peak). CMAP measurements assessed attack severity and duration. Nine participants completed both visits. CMAP percentage of peak amplitudes in the bumetanide (40.6 %) versus placebo (34.9 %) group at 1hr following treatment did not differ significantly (estimated effect difference 5.9 % (95 % CI: (-5.7 %; 17.5 %), p = 0.27, primary outcome). CMAP amplitudes assessed by the area under the curve for early (0-2hr post-treatment) and late (2-4 h post-treatment) efficacy were not statistically different between bumetanide and placebo (early effect estimate 0.043, p = 0.3; late effect estimate 0.085, p = 0.1). Two participants recovered from the attack following bumetanide intake; none recovered following placebo. Bumetanide was well tolerated but not efficacious to rescue a focal attack in an immobilised hand in the majority of patients, although data supports further studies of this agent.
摘要：
这项研究的目的是确定布美他尼是否可以中止HypoPP患者的急性虚弱发作。这是一个随机的，双盲,cross-over,安慰剂对照II期临床试验。通过ADM的等距运动然后休息（McManis方案）引起局灶性弱点发作。参与者进行了两次研究访问，并在发作时接受了安慰剂或2mg布美他尼（定义为外展人的极小值CMAP振幅从峰值下降40％）。CMAP测量评估攻击严重性和持续时间。9名参与者完成了两次访问。治疗后1小时，布美他尼（40.6%）与安慰剂（34.9%）组的CMAP峰值振幅百分比没有显着差异（估计效果差异为5.9%（95%CI：（-5.7%；17.5%），p=0.27，主要结果）。通过早期（治疗后0-2小时）和晚期（治疗后2-4小时）疗效的曲线下面积评估的CMAP振幅在布美他尼和安慰剂之间没有统计学差异（早期疗效估计值0.043，p=0.3；晚期疗效估计值0.085，p=0.1）。两名参与者在摄入布美他尼后从发作中恢复过来；安慰剂后没有人康复。在大多数患者中，布美他尼的耐受性良好，但对挽救固定手的局灶性发作无效。尽管数据支持对这种药物的进一步研究。
