Daily salbutamol in young patients with SMA type II

Marika Pane; Susanna Staccioli; Sonia Messina; Adele D'Amico; Marco Pelliccioni; Elena S. Mazzone; Marina Cuttini; Paolo Alfieri; Roberta Battini; Marion Main; Francesco Muntoni; Enrico Bertini; Marcello Villanova; Eugenio Mercuri

doi:10.1016/j.nmd.2008.05.004

Daily salbutamol in young patients with SMA type II

Marika Pane, Susanna Staccioli, Sonia Messina, Adele D'Amico, Marco Pelliccioni, Elena S. Mazzone, Marina Cuttini, Paolo Alfieri, Roberta Battini, Marion Main, Francesco Muntoni, Enrico Bertini, Marcello Villanova, Eugenio Mercuri

Research output: Contribution to journal › Article › peer-review

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.

Original language	English
Pages (from-to)	536-540
Number of pages	5
Journal	Neuromuscular Disorders
Volume	18
Issue number	7
DOIs	https://doi.org/10.1016/j.nmd.2008.05.004
Publication status	Published - Jul 2008

Keywords

β2 Agonists
Children
Salbutamol
Spinal muscular atrophy
Therapy

ASJC Scopus subject areas

Clinical Neurology
Pediatrics, Perinatology, and Child Health
Developmental Neuroscience
Neurology

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10.1016/j.nmd.2008.05.004

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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.",

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author = "Marika Pane and Susanna Staccioli and Sonia Messina and Adele D'Amico and Marco Pelliccioni and Mazzone, {Elena S.} and Marina Cuttini and Paolo Alfieri and Roberta Battini and Marion Main and Francesco Muntoni and Enrico Bertini and Marcello Villanova and Eugenio Mercuri",

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doi = "10.1016/j.nmd.2008.05.004",

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AU - Pane, Marika

AU - Staccioli, Susanna

AU - Messina, Sonia

AU - D'Amico, Adele

AU - Pelliccioni, Marco

AU - Mazzone, Elena S.

AU - Cuttini, Marina

AU - Alfieri, Paolo

AU - Battini, Roberta

AU - Main, Marion

AU - Muntoni, Francesco

AU - Bertini, Enrico

AU - Villanova, Marcello

AU - Mercuri, Eugenio

PY - 2008/7

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N2 - 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.

AB - 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.

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KW - Spinal muscular atrophy

KW - Therapy

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Daily salbutamol in young patients with SMA type II

Abstract

Keywords

ASJC Scopus subject areas

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