title: Pilot trial of salbutamol in central core and multi-minicore diseases.
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.
authors.affiliation: Dubowitz Neuromuscular Centre, Department of Paediatrics, Imperial College, Hammersmith Hospital, London, UK.
authors.forename: S
authors.initials: S
authors.lastname: Messina
authors.forename: L
authors.initials: L
authors.lastname: Hartley
authors.forename: M
authors.initials: M
authors.lastname: Main
authors.forename: M
authors.initials: M
authors.lastname: Kinali
authors.forename: H
authors.initials: H
authors.lastname: Jungbluth
authors.forename: F
authors.initials: F
authors.lastname: Muntoni
authors.forename: E
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000293:Adolescent; D000318:Adrenergic beta-Agonists / Q000008:administration & dosage*; D000328:Adult; D000420:Albuterol / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D018482:Muscle, Skeletal / Q000503:physiopathology; D020512:Myopathy, Central Core / Q000188:drug therapy* / Q000503:physiopathology; D010865:Pilot Projects; D016059:Range of Motion, Articular / Q000502:physiology; D020127:Recovery of Function / Q000502:physiology
chemical_list: D000318:Adrenergic beta-Agonists; D000420:Albuterol