Pilot trial of albuterol in facioscapulohumeral muscular dystrophy. FSH-DY Group.

Abstract

BACKGROUND/OBJECTIVE:

Facioscapulohumeral muscular dystrophy (FSHD) is currently untreatable, and there have been few therapeutic trials of any agent in the disease. Animal studies have demonstrated that beta2-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 beta2-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 beta2-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.

• Authors: J T Kissel, M P McDermott, R Natarajan, J R Mendell, S Pandya, W M King, R C Griggs, R Tawil
• Journal: Neurology (Neurology) Vol. 50 Issue 5 Pg. 1402-6 (May 1998) ISSN: 0028-3878 [Print] United States
• PMID: 9595995 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)

Chemical References

• Adrenergic beta-Agonists
• Albuterol

Topics

• Absorptiometry, Photon
• Adolescent
• Adrenergic beta-Agonists (therapeutic use)
• Adult
• Albuterol (therapeutic use)
• Body Weight (drug effects)
• Female
• Humans
• Male
• Middle Aged
• Muscular Dystrophies (classification, drug therapy)
• Pilot Projects