title: Periodic salbutamol in facioscapulohumeral muscular dystrophy: a randomized controlled trial.
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).
  
  
  METHODS
  Placebo-controlled double-blind randomized study.
  
  
  METHODS
  Three clinical centers involved in neuromuscular disorders.
  
  
  METHODS
  Ambulatory patients (N=112), 56 per group, with genetically confirmed FSHD, age 18 to 60 years.
  
  
  METHODS
  Salbutamol (sustained released formulation) administered orally at a daily dose of 16 mg using a periodic dosage regimen (3 wks on, 1 wk off).
  
  
  METHODS
  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.
authors: Institute of Myology, Hospital Pitié-Salpêtrière, Paris, France. c.payan@institut-myologie.org
authors: C A
authors: CA
authors: Payan
authors: J Y
authors: JY
authors: Hogrel
authors: E H
authors: EH
authors: Hammouda
authors: L
authors: L
authors: Lacomblez
authors: G
authors: G
authors: Ollivier
authors: V
authors: V
authors: Doppler
authors: B
authors: B
authors: Eymard
authors: S
authors: S
authors: Attarian
authors: J
authors: J
authors: Pouget
authors: C
authors: C
authors: Desnuelle
authors: P
authors: P
authors: Laforêt
mesh_terms: D000293:Adolescent; D000318:Adrenergic beta-Agonists / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D000328:Adult; D000420:Albuterol / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001794:Blood Pressure / Q000187:drug effects; D003692:Delayed-Action Preparations; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005260:Female; D006339:Heart Rate / Q000187:drug effects; D006801:Humans; D008297:Male; D008875:Middle Aged; D053580:Muscle Strength / Q000187:drug effects*; D020391:Muscular Dystrophy, Facioscapulohumeral / Q000188:drug therapy* / Q000503:physiopathology; D055815:Young Adult
chemical_list: D000318:Adrenergic beta-Agonists; D003692:Delayed-Action Preparations; D000420:Albuterol