title: Suitability of North Star Ambulatory Assessment in young boys with Duchenne muscular dystrophy.
abstract: The aim of this study was to establish the suitability of the North Star Ambulatory Assessment for use in young boys with Duchenne muscular dystrophy. We studied 147 typically developing and 144 boys affected by Duchenne muscular dystrophy between the ages of 3 and 5 years. More than 85% of the typically developing boys by the age of 4 years had full scores on all the items with total scores ≥33/34. Before the age of 4 years more than 15% of the typically developing boys did not achieve full scores on all the items. Some items, such as standing on one leg, showed significant improvement with age. In contrast, other activities were rarely achieved even in the older boys. Even if there was a progressive increase in scores with age, both total and individual item scores in Duchenne were still far from those obtained in the typically developing children of the same age. Our findings suggest that the North Star Ambulatory Assessment can be reliably used at least from the age of 4 years. Longitudinal natural history data studies are needed to assess possible changes over time and the possible effect of early steroids.
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Sivo
authors.affiliation: Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
authors.forename: Valeria
authors.initials: V
authors.lastname: Ricotti
authors.affiliation: Developmental Neurology Unit, C. Besta Neurological Institute IRCCS, Milan, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Baranello
authors.affiliation: Department of Developmental Neuroscience, IRCSS Stella Maris, Pisa, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Frosini
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Mazzone
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Flaviana
authors.initials: F
authors.lastname: Bianco
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Lavinia
authors.initials: L
authors.lastname: Fanelli
authors.affiliation: Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
authors.forename: Marion
authors.initials: M
authors.lastname: Main
authors.affiliation: Developmental Neurology Unit, C. Besta Neurological Institute IRCCS, Milan, Italy.
authors.forename: Alice
authors.initials: A
authors.lastname: Corlatti
authors.affiliation: Unit of Neuromuscular and Neurodegenerative diseases, Department of Neurosciences, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.affiliation: Unit of Neuromuscular and Neurodegenerative diseases, Department of Neurosciences, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Giulia
authors.initials: G
authors.lastname: Colia
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Scalise
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Concetta
authors.initials: C
authors.lastname: Palermo
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Alfonsi
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Giovanna
authors.initials: G
authors.lastname: Tritto
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Domenico M
authors.initials: DM
authors.lastname: Romeo
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Graziano
authors.affiliation: Department of Developmental Neuroscience, IRCSS Stella Maris, Pisa, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Battini
authors.affiliation: Developmental Neurology Unit, C. Besta Neurological Institute IRCCS, Milan, Italy.
authors.forename: Lucia
authors.initials: L
authors.lastname: Morandi
authors.affiliation: Unit of Neuromuscular and Neurodegenerative diseases, Department of Neurosciences, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.affiliation: Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy. Electronic address: eumercuri@gmail.com.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D002675:Child, Preschool; D005080:Exercise Test / Q000379:methods*; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis*; D011446:Prospective Studies; D012720:Severity of Illness Index
keywords: Duchenne muscular dystrophy; North Star Ambulatory Assessment; Outcome measure; Typically developing boys