Double-Blind, Randomized, Controlled Trial of Albuterol in Facioscapulohumeral Dystrophy

Abstract

John T. Kissel, Columbus, OH; Rabi Tawil, Michael McDermott, Rochester, NY; Wendy M. King, Columbus, OH; Shree Pandya, Rochester, NY; Jerry R. Mendell, Columbus, OH; Robert Griggs, Rochester, NY; and the FSH-DY Group. Objective: This study was designed as a 1-year, prospective, randomized, double-blind, placebo-controlled trial of albuterol, a β2-adrenergic agonist, in facioscapulohumeral muscular dystrophy (FSHD). Background: The gene for FSHD has not been cloned and there is currently no treatment. The β2-agonists have an anabolic effect that has been shown to increase strength in healthy adults. A preliminary open-label trial involving 15 FSHD patients suggested that albuterol might increase muscle mass and strength in patients with this disease. Design/Methods: Ninety ambulatory adult FSHD patients (49 men, 41 women, ages 18 to 57 years) were randomized to one of three groups (30 patients each). Two groups took sustained-release albuterol (Proventil Repetabs, Schering Corporation) at a dose of either 8.0 mg or 16 mg every 12 hours; the third group took identical placebo. All patients took the same number of pills per day. Assessments were performed at weeks 4, 13, 26, and 52 after enrollment. The primary outcome measure was the change from baseline to week 52 in the composite (12 tested muscles) computerized maximum voluntary isometric contraction testing (MVICT) score, measured as the number of standard deviation units from normal. A secondary outcome was the mean change over the same period in the average muscle score (AMS) assessed through manual muscle testing (MMT) of 38 muscle groups. Patients, investigators, and clinical evaluators were blinded to treatment status. The study was designed to detect a mean composite MVICT change of 0.82, or mean AMS change of 0.20, with a power of 80%. Results: Eighty-four patients completed the study. The mean changes in composite MVICT scores were not …