title: Perceived efficacy of salbutamol by persons with spinal muscular atrophy: A mixed methods study.
abstract: The aim of this study was to assess the perceived effect of salbutamol in adult patients with spinal muscular atrophy and to evaluate the usefulness of the World Health Organization Disability Assessment Schedule II (WHODAS II) and Fatigue Severity Scale (FSS) for its measurement.
  
  
  
  A longitudinal mixed methods study was performed. Ten patients were interviewed and completed WHODAS II and FSS questionnaires to assess disability and fatigue at 2 time-points. Inductive thematic analysis was used for qualitative data. The non-parametric Wilcoxon test was performed for quantitative analysis.
  
  
  
  All participants reported an improvement in their condition after salbutamol consumption. WHODAS II and FSS reliably captured changes in patients' disability and fatigue.
  
  
  
  The mixed methods design allowed us to identify the functional domains in which participants experienced effects of salbutamol. Patients were satisfied with the treatment as shown by decreased fatigue, improved functioning, and infrequent side effects. Muscle Nerve, 2016 Muscle Nerve 54: 843-849, 2016.
authors.affiliation: Department of Neuroimmunology and Neuromuscular Diseases, SOD Day Hospital/Day Service, Neurological Institute C. Besta IRCCS Foundation, Via Celoria 11, 20133, Milan, Italy. ambra.giovannetti@istituto-besta.it.
authors.forename: Ambra M
authors.initials: AM
authors.lastname: Giovannetti
authors.affiliation: Department of Neuroimmunology and Neuromuscular Diseases, SOD Day Hospital/Day Service, Neurological Institute C. Besta IRCCS Foundation, Via Celoria 11, 20133, Milan, Italy.
authors.forename: Maria Barbara
authors.initials: MB
authors.lastname: Pasanisi
authors.affiliation: Department of Neurosurgery, Radiotherapy Unit, Neurological Institute C. Besta IRCCS Foundation, Milan, Italy.
authors.forename: Milda
authors.initials: M
authors.lastname: Černiauskaitė
authors.affiliation: Department of Developmental Neurology, Neurological Institute C. Besta IRCCS Foundation, Milan, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Bussolino
authors.affiliation: Neurology, Public Health and Disability Unit, Neurological Institute C. Besta IRCCS Foundation, Milan, Italy.
authors.forename: Matilde
authors.initials: M
authors.lastname: Leonardi
authors.affiliation: Department of Neuroimmunology and Neuromuscular Diseases, SOD Day Hospital/Day Service, Neurological Institute C. Besta IRCCS Foundation, Via Celoria 11, 20133, Milan, Italy.
authors.forename: Lucia
authors.initials: L
authors.lastname: Morandi
mesh_terms: D000328:Adult; D000420:Albuterol / Q000627:therapeutic use*; D001993:Bronchodilator Agents / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy*; D012720:Severity of Illness Index; D018709:Statistics, Nonparametric; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D001993:Bronchodilator Agents; D000420:Albuterol
keywords: disability; fatigue; mixed methods study; patient-reported outcome; qualitative study; salbutamol