Bradley W G, Badger G J, Tandan R, Fillyaw M J, Young J, Fries T J, Krusinski P B, Witarsa M, Boerman J, Blair C J
Department of Neurology, University of Vermont, Burlington.
Neurology. 1988 Nov;38(11):1731-9. doi: 10.1212/wnl.38.11.1731.
We report three 12-month, double-blind, three-phase studies comparing the effect of placebo and 40 mg and 100 mg IM daily of purified bovine brain gangliosides (Cronassial) in chronic neuromuscular diseases. Thirty patients with Charcot-Marie-Tooth disease, 16 with idiopathic polyneuropathy, and 30 with spinocerebellar degeneration had neuromuscular function measured monthly by quantitative testing of motor and sensory function, coordination, and electrophysiologic factors. Analysis of these studies, and of longer term (up to 2 years) open studies of 100 mg daily of Cronassial in 67 patients failed to show therapeutic efficacy of Cronassial. Statistical power calculations indicated that five of the 37 measures had greater than a 70% chance of detecting a 20% difference in the rate of progression of the active-drug and placebo groups. A number of measures significantly improved during prolonged placebo treatment, suggesting that the placebo effect has a strong influence on "objective" measures of neuromuscular function.
我们报告了三项为期12个月的双盲、三阶段研究，比较了安慰剂以及每日40毫克和100毫克肌肉注射纯化牛脑苷脂（克瑙斯亚尔）对慢性神经肌肉疾病的影响。30例夏科-马里-图思病患者、16例特发性多发性神经病患者和30例脊髓小脑变性患者，通过对运动和感觉功能、协调性及电生理因素进行定量测试，每月测量一次神经肌肉功能。对这些研究以及对67例患者每日服用100毫克克瑙斯亚尔的长期（长达2年）开放性研究进行分析，均未显示克瑙斯亚尔具有治疗效果。统计功效计算表明，37项测量指标中有5项有超过70%的可能性检测到活性药物组和安慰剂组在进展速率上存在20%的差异。在长时间的安慰剂治疗期间，多项测量指标有显著改善，这表明安慰剂效应对神经肌肉功能的“客观”测量有很大影响。
