Daily salbutamol in young patients with SMA type II.
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.
Pane
Marika
M
Department of Paediatric Neurology, Catholic University, Policlinico Gemelli Largo Gemelli, 00168 Rome, Italy.
Staccioli
Susanna
S
Messina
Sonia
S
D'Amico
Adele
A
Pelliccioni
Marco
M
Mazzone
Elena S
ES
Cuttini
Marina
M
Alfieri
Paolo
P
Battini
Roberta
R
Main
Marion
M
Muntoni
Francesco
F
Bertini
Enrico
E
Villanova
Marcello
M
Mercuri
Eugenio
E
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
D000318:Adrenergic beta-Agonists; D000420:Albuterol