Sleep Med. 2004 Mar;5(2):119-23. doi: 10.1016/j.sleep.2003.11.002.
This study was conducted to demonstrate the long-term efficacy of sodium oxybate for the long-term treatment of cataplexy in patients with narcolepsy.
Fifty-five (55) narcoleptic patients with cataplexy who had received continuous treatment with sodium oxybate for 7-44 months (mean 21 months) were enrolled in a double-blind treatment withdrawal paradigm. A 2-week single-blind sodium oxybate treatment phase established a baseline for the weekly occurrence of cataplexy. This was followed by a 2-week double-blind phase in which patients were randomized to receive unchanged drug therapy or placebo. Patients recorded the incidence of cataplexy attacks and adverse events in daily diaries.
During the 2-week double-blind phase, the abrupt cessation of sodium oxybate therapy in the placebo patients resulted in a significant increase in the number of cataplexy attacks (median=21; P<0.001 ) compared to patients who remained on sodium oxybate (median=0). Cataplexy attacks returned gradually with placebo patients reporting a median of 4.2 and 11.7 cataplexy attacks during the first and second weeks, respectively. There were no symptoms of frank withdrawal.
This controlled trial provides evidence supporting the long-term efficacy of sodium oxybate for the treatment of cataplexy. In contrast with antidepressant drug therapy, there is no evidence of rebound cataplexy upon abrupt discontinuation of treatment.
本研究旨在证明羟丁酸钠对发作性睡病患者猝倒症的长期治疗效果。
55例患有猝倒症的发作性睡病患者接受羟丁酸钠持续治疗7 - 44个月（平均21个月），参与双盲治疗撤药模式研究。为期2周的单盲羟丁酸钠治疗阶段确定了每周猝倒发作情况的基线。随后是为期2周的双盲阶段，在此阶段患者被随机分配接受不变的药物治疗或安慰剂治疗。患者在日常日记中记录猝倒发作的发生率和不良事件。
在为期2周的双盲阶段，与继续接受羟丁酸钠治疗的患者（中位数 = 0）相比，安慰剂组患者突然停止羟丁酸钠治疗导致猝倒发作次数显著增加（中位数 = 21；P < 0.001）。安慰剂组患者的猝倒发作逐渐恢复，在第一周和第二周分别报告中位数为4.2次和11.7次猝倒发作。没有明显的撤药症状。
这项对照试验提供了证据支持羟丁酸钠治疗猝倒症的长期疗效。与抗抑郁药物治疗不同，没有证据表明突然停药会出现猝倒症反弹。
