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).

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 16 mg using a periodic dosage regimen (3 wks on, 1 wk 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 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: C A Payan, J Y Hogrel, E H Hammouda, L Lacomblez, G Ollivier, V Doppler, B Eymard, S Attarian, J Pouget, C Desnuelle, P Laforêt
• Journal: Archives of physical medicine and rehabilitation (Arch Phys Med Rehabil) Vol. 90 Issue 7 Pg. 1094-101 (Jul 2009) ISSN: 1532-821X [Electronic] United States
• PMID: 19577021 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Adrenergic beta-Agonists
• Delayed-Action Preparations
• Albuterol

Topics

• Adolescent
• Adrenergic beta-Agonists (administration & dosage, adverse effects, therapeutic use)
• Adult
• Albuterol (administration & dosage, adverse effects, therapeutic use)
• Blood Pressure (drug effects)
• Delayed-Action Preparations
• Double-Blind Method
• Drug Administration Schedule
• Female
• Heart Rate (drug effects)
• Humans
• Male
• Middle Aged
• Muscle Strength (drug effects)
• Muscular Dystrophy, Facioscapulohumeral (drug therapy, physiopathology)
• Young Adult