Sonia Khirani1, Ivana Dabaj2, Alessandro Amaddeo3, Jorge Olmo Arroyo4, Jacques Ropers5, Stéphane Tirolien6, Véronique Coudert6, Brigitte Estournet2, Brigitte Fauroux3, Susana Quijano-Roy7. 1. ASV Santé, Gennevilliers, France; AP-HP, Hôpital Necker-Enfants Malades, Pediatric Noninvasive Ventilation and Sleep Unit, Paris, France. 2. AP-HP, Hôpital Raymond Poincaré, Hôpitaux Universitaires Paris-Ile-de-France Ouest, Pôle pédiatrique, Service de Pédiatrie, Garches, France; Centre de Référence Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Garches, France; Réseau National de Maladies Neuromusculaires FILNEMUS, Garches, France. 3. AP-HP, Hôpital Necker-Enfants Malades, Pediatric Noninvasive Ventilation and Sleep Unit, Paris, France; INSERM U955, Créteil, France; Paris Descartes University, Paris, France. 4. AP-HP, Hôpital Necker-Enfants Malades, Pediatric Noninvasive Ventilation and Sleep Unit, Paris, France. 5. Hôpitaux Universitaires Paris Île-de-France Ouest, Unité de Recherche Clinique, Boulogne, France. 6. AP-HP, Hôpital Raymond Poincaré, Hôpitaux Universitaires Paris-Ile-de-France Ouest, Pôle pédiatrique, Service de Pédiatrie, Garches, France. 7. AP-HP, Hôpital Raymond Poincaré, Hôpitaux Universitaires Paris-Ile-de-France Ouest, Pôle pédiatrique, Service de Pédiatrie, Garches, France; Centre de Référence Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye (GNMH), Garches, France; Réseau National de Maladies Neuromusculaires FILNEMUS, Garches, France; INSERM U1179, Université Versailles Saint-Quentin (UVSQ), UFR des sciences de la santé Simone Veil, Montigny, France. Electronic address: susana.quijano-roy@aphp.fr.
Abstract
BACKGROUND: Oral salbutamol has shown clinical benefits in spinal muscular atrophy (SMA). We studied its effect on the respiratory muscle strength in children with different types of SMA. METHODS: Lung and respiratory muscle functions were assessed in children receiving daily oral salbutamol for at least one year. The respiratory data of age-matched SMA II historical control subjects were compared with data of SMA II patients receiving salbutamol. RESULTS: Seven children (6.4 ± 2.0 years old, range four to ten; one SMA I, five SMA II, and one SMA III) treated with salbutamol (duration 23 ± 8 months) were assessed. Maximal static inspiratory pressure, sniff nasal inspiratory pressure, and slow vital capacity were significantly better in the salbutamol-treated SMA II group compared with control subjects (P < 0.05). CONCLUSIONS: Long-term oral salbutamol showed benefits in respiratory function in children with SMA and appeared to increase the strength of the inspiratory muscles in a small cohort of SMA II patients.
BACKGROUND: Oral salbutamol has shown clinical benefits in spinal muscular atrophy (SMA). We studied its effect on the respiratory muscle strength in children with different types of SMA. METHODS: Lung and respiratory muscle functions were assessed in children receiving daily oral salbutamol for at least one year. The respiratory data of age-matched SMA II historical control subjects were compared with data of SMA II patients receiving salbutamol. RESULTS: Seven children (6.4 ± 2.0 years old, range four to ten; one SMA I, five SMA II, and one SMA III) treated with salbutamol (duration 23 ± 8 months) were assessed. Maximal static inspiratory pressure, sniff nasal inspiratory pressure, and slow vital capacity were significantly better in the salbutamol-treated SMA II group compared with control subjects (P < 0.05). CONCLUSIONS: Long-term oral salbutamol showed benefits in respiratory function in children with SMA and appeared to increase the strength of the inspiratory muscles in a small cohort of SMA II patients.
Authors: Renske I Wadman; W Ludo van der Pol; Wendy Mj Bosboom; Fay-Lynn Asselman; Leonard H van den Berg; Susan T Iannaccone; Alexander Fje Vrancken Journal: Cochrane Database Syst Rev Date: 2020-01-06