Pilot trial of salbutamol in central core and multi-minicore diseases

Neuropediatrics. 2004 Oct;35(5):262-6. doi: 10.1055/s-2004-821173.

Abstract

Several studies have documented positive effects of beta-adrenergic agonists on human skeletal muscle with regard to muscle mass and strength. The aim of this pilot study was to evaluate the effect of the beta2-agonist salbutamol (albuterol) in a group of children with central core disease and multi-minicore disease. Thirteen patients, 8 with central core disease (mean age 17.5 years) and 5 with minicore disease (mean age 13.6 years) received oral salbutamol at a dose of 2 mg four times a day. Measures of efficacy were the change from baseline at 3 and 6 months in muscle strength, assessed by MRC score, myometry, functional measures and forced vital capacity. Statistical analysis was performed using repeated measures ANOVA (significance level < 0.05). Two patients with central core disease stopped the medication after one month because they did not notice any improvement and another one with minicore disease after 4 months because of increased tremors and palpitations. The remaining ten (6 with central core and 4 with minicore disease) completed the course of salbutamol without any significant adverse effects. There were significant increases in myometry, MRC scores and forced vital capacity between baseline and the six-month assessments. For both myometry and MRC the difference was already significant at 3 months and this was associated with a significant increase in functional abilities assessed with a structured functional scale. Our results suggest that salbutamol was overall well tolerated and might be beneficial in both central core and minicore patients. Larger prospective randomised, double-blind, placebo-controlled trials with salbutamol will be needed to confirm these preliminary findings.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adrenergic beta-Agonists / administration & dosage*
  • Adult
  • Albuterol / administration & dosage*
  • Child
  • Child, Preschool
  • Cohort Studies
  • Drug Administration Schedule
  • Female
  • Humans
  • Male
  • Middle Aged
  • Muscle, Skeletal / physiopathology
  • Myopathy, Central Core / drug therapy*
  • Myopathy, Central Core / physiopathology
  • Pilot Projects
  • Range of Motion, Articular / physiology
  • Recovery of Function / physiology

Substances

  • Adrenergic beta-Agonists
  • Albuterol