Randomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy
Randomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy
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DOI：
10.1212/wnl.57.8.1434
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发表时间：
2001-10-23
期刊：
影响因子：
9.9
通讯作者：
Kolassa, J
中科院分区：
文献类型：
--
作者：
Kissel, JT;McDermott, MP;Kolassa, J
背景/目的：动物和人类研究表明，β(2)-肾上腺素能激动剂对肌肉发挥合成代谢作用，诱导和预防各种侮辱后的萎缩。根据沙丁胺醇在15名面肩肩关节营养不良(FSHD)患者中的开放试验数据，作者进行了一项随机、双盲、安慰剂对照的沙丁胺醇缓释试验。方法：90例患者随机分为3组：安慰剂组、沙丁胺醇8.0 mg每日两次或沙丁胺醇16.0 mg每日两次。患者在基线和第13、26、52周接受为期一年的评估。主要结果是通过最大自愿等长收缩测试(MVICT)获得52周的全球力量变化。次要结果包括52周时通过手动肌肉测试(MMT)、握力、功能测试和双能X射线吸收测量仪(DEXA)评估的肌肉质量的力量变化。结果：84例患者完成研究。两组间MVICT综合评分的平均变化无显著差异(平均+/-SD：安慰剂0.20+/-0.91；低剂量-0.04+/-0.84；大剂量0.08+/-0.98)。同样，平均MMT变化没有差异(安慰剂0.04+/-0.16；低剂量-0.03+/-0.13；高剂量0.00+/-0.15)。与安慰剂(安慰剂-0.53+/-4.13，低剂量+1.90+/-3.34[p=0.02]，高剂量+1.70+/-4.13[p=0.03])相比，两个治疗组的握力都有所改善。服用地塞米松的高剂量组瘦体重(+1.57+/-1.71千克)显著高于安慰剂组(0.25+/-2.24千克；p=0.007)。沙丁胺醇耐受性良好；副作用包括抽筋、震颤、失眠和紧张。结论：尽管沙丁胺醇没有改善FSHD患者的整体力量或功能，但它确实增加了肌肉质量并改善了一些力量指标。
Background/Objectives: Animal and human studies suggest that beta (2)-adrenergic agonists exert anabolic effects on muscles, inducing and preventing atrophy after a variety of insults. Based on data from an open-label trial of albuterol in 15 patients with facioscapulohumeral dystrophy (FSHD), the authors conducted a randomized, double-blind, placebo-controlled trial of sustained-release albuterol in this disease. Methods: Ninety patients were randomized to three groups: placebo; 8.0 mg albuterol twice daily; or 16.0 mg albuterol twice daily. Patients were treated for 1 year with assessments at baseline and weeks 13, 26, and 52. The primary outcome was the 52-week change in global strength by maximum voluntary isometric contraction testing (MVICT). Secondary outcomes included changes at 52 weeks in strength by manual muscle testing (MMT), grip strength, functional testing, and muscle mass assessed by dual energy x-ray absorptiometry (DEXA). Results: Eighty-four patients completed the study. The mean changes in composite MVICT scores were not significantly different between the groups (mean +/- SD: placebo 0.20 +/- 0.91; low dose -0.04 +/- 0.84; high dose 0.08 +/- 0.98). Similarly, there were no differences in the mean MMT change (placebo 0.04 +/- 0.16; low dose -0.03 +/- 0.13; high dose 0.00 +/- 0.15). Grip improved in both treatment groups compared to placebo (placebo -0.53 +/- 4.13, low dose +1.90 +/- 3.34 [p = 0.02], high dose +1.70 +/- 4.13 [p = 0.03]). The high-dose group had a significant increase in lean mass by DEXA (+1.57 +/- 1.71 kg) compared to placebo (0.25 +/- 2.24; p = 0.007). Albuterol was well tolerated; side effects included cramps, tremors, insomnia, and nervousness. Conclusions: Although albuterol did not improve global strength or function in patients with FSHD, it did increase muscle mass and improve some measures of strength.