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

Messina, S; Hartley, L; Main, M; Kinali, M; Jungbluth, H; Muntoni, F; Mercuri, E; (2004) Pilot trial of salbutamol in central core and multi-minicore diseases. NEUROPEDIATRICS , 35 (5) 262 - 266. 10.1055/s-2004-821173.

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Abstract

Several studies have documented positive effects of P-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.

Type: Article
Title: Pilot trial of salbutamol in central core and multi-minicore diseases
DOI: 10.1055/s-2004-821173
Keywords: beta-agonists, skeletal muscle, central core disease, multi-minicore disease, therapeutic trial, SPINAL MUSCULAR-ATROPHY, SKELETAL-MUSCLE, CLENBUTEROL, DYSTROPHY, ALBUTEROL, STRENGTH, CHILDREN, MYOPATHY, AGONIST, GENE
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health > ICH Developmental Neurosciences Prog
URI: http://discovery.ucl.ac.uk/id/eprint/91489
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