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, Laforêt P. 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).

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 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.

Key Words: Clinical trial, Phase III, Facioscapulohumeral muscular dystrophy, Pharmacokinetics, Rehabilitation, Salbutamol

List of Abbreviations: ECG, electrocardiogram, FSHD, facioscapulohumeral muscular dystrophy, MMT, manual muscle testing, MVIC, maximum voluntary isometric contractions, QMT, quantitative muscle testing, VAS, visual analog scale

a Institute of Myology, Hospital Pitié-Salpêtrière, Paris, France

b Association Française contre les Myopathies (AFM), Evry, France

c Department of Neurology, Hospital Pitié-Salpêtrière, AP-HP; University Pierre & Marie Curie UPMC Paris 6; INSERM UMR 678; Paris, France

d Reference Center of Neuromuscular Disorders Paris-Est, Hospital Pitié-Salpêtrière, AP-HP; University Pierre & Marie Curie UPMC Paris 6; Paris, France

e Reference Center for Neuromuscular Disorders & Amyotrophic Lateral Sclerosis, University Hospital La Timone, AP-HM, Marseille, France

f Reference Center for Neuromuscular Disorders & Amyotrophic Lateral Sclerosis, Hospital l'Archet, Nice, France

g Reference Center of Neuromuscular Disorders Paris-Est, Hospital Pitié-Salpêtrière, AP-HP, Paris, France

Correspondence to C.A. Payan, MD, Institut de Myologie, Groupe Hospitalier Pitié-Salpêtrière, Batiment Babinski, 47 bd de l'Hôpital, 75651 Paris Cedex 13, France

Supported by the Association Française contre les Myopathies (AFM grant no. 6742) and sponsored by the Assistance Publique Hôpitaux de Paris (AP-HP, PHRC grant no. AOM98125).

No commercial party having a direct financial interest in the results of the research supporting this article has or will confer a benefit on the authors or on any organization with which the authors are associated.

Reprints are not available from the author.

PII: S0003-9993(09)00208-1

doi:10.1016/j.apmr.2008.12.027

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