Article

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.

Langue : ANGLAIS