ohio universitesinde pilot deney yapılmış. 15 tane fsh hastasına 3 ay boyunca günde 16 mg albuterol isimli ilaç vermişler. Kas kütlelerinde % 12 lik bir artış olmuş. Uzmanlar görüş birliğine varmışlar. Albuterol bir astım ilacı. Fazla bir yan etkisi yok. Türkiye'de yok. Bulursam kullanıcam.
Diğer kas hastalıgı tiplerinde de işe yarama ihtimali varmış.
Pilot trial of albuterol in facioscapulohumeral muscular dystrophy
J. T. Kissel, MD, M. P. McDermott, PhD, R. Natarajan, MA, J. R. Mendell, MD, S. Pandya, MS, W. M. King, PT, R. C. Griggs, MD, R. Tawil, MD and The FSH-DY Group*
From the Department of Neurology (Drs. Kissel and Mendell, and W.M. King), Ohio State University, Columbus, OH; and the Department of Neurology and the Wayne C. Gorrell Jr. Molecular Biology Laboratory (Drs. Tawil, McDermott, and Griggs, and S. Pandya) and the Department of Biostatistics (Dr. McDermott and R. Natarajan), University of Rochester School of Medicine and Dentistry, NY.
Address correspondence and reprint requests to Dr. John T. Kissel, Department of Neurology, Division of Neuromuscular Disease, The Ohio State University Medical Center, 1654 Upham Drive, Columbus, OH 43210.
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 ß2-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 ß2-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 ß2-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.