Pilot trial of albuterol in facioscapulohumeral muscular dystrophy
Pilot trial of albuterol in facioscapulohumeral muscular dystrophy
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DOI：
10.1212/wnl.50.5.1402
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发表时间：
1998-05-01
期刊：
影响因子：
9.9
通讯作者：
Tawil, R
中科院分区：
文献类型：
--
作者：
Kissel, JT;McDermott, MP;Tawil, R
Background/Objective: Facioscapulohumeral muscular dystrophy (FSBD) is currently untreatable, and there have been few therapeutic trials of any agent in the disease. Animal studies have demonstrated that beta(2)-adrenergic agonists induce muscle hypertrophy and prevent atrophy after a variety of physical and biochemical insults, and two human studies have shown that these agents increase certain measures of strength in healthy volunteers. We conducted an open-label pilot trial of a beta(2)-agonist (albuterol) in patients with FSHD. Methods: Fifteen FSHD patients were given sustained-release albuterol (16.0 mg/day) for 3 months. The primary outcome measure was lean body mass, which was assessed through dual energy X-ray absorptiometry (DEXA). Strength was evaluated through maximal voluntary isometric contraction testing (MVICT) and manual muscle testing. Results: Albuterol significantly increased DEXA lean body mass (the skeletal muscle compartment) by 1.29 +/- 1.18 kg (mean +/- SD, p = 0.001). Strength assessed through composite MVICT scores also increased by an average of 0.33 +/- 0.60 (p = 0.05), representing an overall 12% improvement in strength. Conclusions: These encouraging results suggest that beta(2)-agonists may have a role in treating FSHD and possibly other neuromuscular diseases. The effects of albuterol in FSHD are currently being evaluated in a larger, randomized, double-blind, placebo-controlled trial lasting 1 year.
