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Arch Phys Med Rehabil. 2009 Jul;90(7):1094-101. doi: 10.1016/j.apmr.2008.12.027.

Periodic salbutamol in facioscapulohumeral muscular dystrophy: a randomized controlled trial.

Author information

1
Institute of Myology, Hospital Pitié-Salpêtrière, Paris, France. c.payan@institut-myologie.org

Abstract

OBJECTIVE:

To evaluate the effects on muscle strength of salbutamol administered for 6 months using a periodic regimen in patients presenting with facioscapulohumeral muscular dystrophy (FSHD).

DESIGN:

Placebo-controlled double-blind randomized study.

SETTING:

Three clinical centers involved in neuromuscular disorders.

PARTICIPANTS:

Ambulatory patients (N=112), 56 per group, with genetically confirmed FSHD, age 18 to 60 years.

INTERVENTIONS:

Salbutamol (sustained released formulation) administered orally at a daily dose of 16 mg using a periodic dosage regimen (3 wks on, 1 wk off).

MAIN OUTCOME MEASURES:

Muscle strength was assessed with quantitative muscle testing (QMT), manual muscle testing (MMT), and timed motor tests. Patients were evaluated at baseline, and 3 and 6 months later. Plasma drug assays were carried out at each visit.

RESULTS:

There was no significant change with periodic use of salbutamol in the total composite QMT z-score, MMT score, or timed motor tests. Salbutamol was well tolerated. Lack of efficacy did not seem to be related to plasma concentrations, which were within the expected range.

CONCLUSIONS:

Results from this study and previous controlled trials preclude at present the use of salbutamol as routine treatment for FSHD, even if we cannot exclude improvement from anabolic effects with a longer duration of treatment.

PMID:
19577021
DOI:
10.1016/j.apmr.2008.12.027
[Indexed for MEDLINE]
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