title: Daily salbutamol in young patients with SMA type II.
abstract: The aim of this open pilot study was to establish the profile of tolerability and clinical response of salbutamol (albuterol) in a cohort of young children affected by type II spinal muscular atrophy (SMA). Twenty-three children between 30 months and 6 years of age were treated with salbutamol (2 mg three times a day) for 1 year. All children were longitudinally assessed using the Hammersmith motor functional scale 6 months before treatment started (T0), at baseline (T1) and 6 and 12 months later. There was no significant change in function between T0 and T1 assessments, but the functional scores recorded after 6 and 12 months of treatment were significantly higher than those recorded at baseline (p=0.006). Our results suggest that salbutamol may be beneficial to SMA patients without producing any major side effect. Larger prospective randomized, double-blind, placebo controlled trials are needed to confirm these preliminary findings.
authors.affiliation: Department of Paediatric Neurology, Catholic University, Policlinico Gemelli Largo Gemelli, 00168 Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.forename: Susanna
authors.initials: S
authors.lastname: Staccioli
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.forename: Marco
authors.initials: M
authors.lastname: Pelliccioni
authors.forename: Elena S
authors.initials: ES
authors.lastname: Mazzone
authors.forename: Marina
authors.initials: M
authors.lastname: Cuttini
authors.forename: Paolo
authors.initials: P
authors.lastname: Alfieri
authors.forename: Roberta
authors.initials: R
authors.lastname: Battini
authors.forename: Marion
authors.initials: M
authors.lastname: Main
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.forename: Marcello
authors.initials: M
authors.lastname: Villanova
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000318:Adrenergic beta-Agonists / Q000627:therapeutic use*; D000420:Albuterol / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004562:Electrocardiography / Q000379:methods; D005260:Female; D006801:Humans; D008297:Male; D010865:Pilot Projects; D012189:Retrospective Studies; D012720:Severity of Illness Index; D014897:Spinal Muscular Atrophies of Childhood / Q000188:drug therapy*; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000318:Adrenergic beta-Agonists; D000420:Albuterol