Periodic Salbutamol in Facioscapulohumeral Muscular Dystrophy: A Randomized Controlled Trial

Payan CA, Hogrel JY, Hammouda EH, Lacomblez L, Ollivier G, Doppler V, Eymard B, Attarian S, Pouget J, Desnuelle C, Laforêt P. Periodic salbutamol in facioscapulohumeral muscular dystrophy: a randomized controlled trial.ObjectiveTo evaluate the effects on muscle strength of salbutamol administered for 6 months using a periodic regimen in patients presenting with facioscapulohumeral muscular dystrophy (FSHD).DesignPlacebo-controlled double-blind randomized study.SettingThree clinical centers involved in neuromuscular disorders.ParticipantsAmbulatory patients (N=112), 56 per group, with genetically confirmed FSHD, age 18 to 60 years.InterventionsSalbutamol (sustained released formulation) administered orally at a daily dose of 16mg using a periodic dosage regimen (3wks on, 1wk off).Main Outcome MeasuresMuscle 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.ResultsThere 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.ConclusionsResults 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.