Periodic Salbutamol in Facioscapulohumeral Muscular Dystrophy: A Randomized Controlled Trial

Abstract Payan CA, Hogrel JY, Hammouda EH, Lacomblez L, Ollivier G, Doppler V, Eymard B, Attarian S, Pouget J, Desnuelle C, Laforet P. Periodic salbutamolin facioscapulohumeral muscular dystrophy: a randomized controlled trial. Objective To evaluate the effects on muscle strength of salbutamoladministered 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 16mg using a periodic dosage regimen (3wks on, 1wk 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 salbutamolin the total composite QMT z-score, MMT score, or timed motor tests. Salbutamolwas 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 salbutamolas routine treatment for FSHD, even if we cannot exclude improvement from anabolic effects with a longer duration of treatment.