Periodic salbutamol in facioscapulohumeral muscular dystrophy: a randomized controlled trial.
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.
Payan
C A
CA
Institute of Myology, Hospital Pitié-Salpêtrière, Paris, France. c.payan@institut-myologie.org
Hogrel
J Y
JY
Hammouda
E H
EH
Lacomblez
L
L
Ollivier
G
G
Doppler
V
V
Eymard
B
B
Attarian
S
S
Pouget
J
J
Desnuelle
C
C
Laforêt
P
P
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
D000318:Adrenergic beta-Agonists; D003692:Delayed-Action Preparations; D000420:Albuterol