title: North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy.
abstract: The North Star Ambulatory Assessment is a functional scale specifically designed for ambulant boys affected by Duchenne muscular dystrophy (DMD). Recently the 6-minute walk test has also been used as an outcome measure in trials in DMD. The aim of our study was to assess a large cohort of ambulant boys affected by DMD using both North Star Assessment and 6-minute walk test. More specifically, we wished to establish the spectrum of findings for each measure and their correlation. This is a prospective multicentric study involving 10 centers. The cohort included 112 ambulant DMD boys of age ranging between 4.10 and 17 years (mean 8.18±2.3 DS). Ninety-one of the 112 were on steroids: 37/91 on intermittent and 54/91 on daily regimen. The scores on the North Star assessment ranged from 6/34 to 34/34. The distance on the 6-minute walk test ranged from 127 to 560.6 m. The time to walk 10 m was between 3 and 15 s. The time to rise from the floor ranged from 1 to 27.5 s. Some patients were unable to rise from the floor. As expected the results changed with age and were overall better in children treated with daily steroids. The North Star assessment had a moderate to good correlation with 6-minute walk test and with timed rising from floor but less with 10 m timed walk/run test. The 6-minute walk test in contrast had better correlation with 10 m timed walk/run test than with timed rising from floor. These findings suggest that a combination of these outcome measures can be effectively used in ambulant DMD boys and will provide information on different aspects of motor function, that may not be captured using a single measure.
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Mazzone
authors.forename: Diego
authors.initials: D
authors.lastname: Martinelli
authors.forename: Angela
authors.initials: A
authors.lastname: Berardinelli
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.forename: Gessica
authors.initials: G
authors.lastname: Vasco
authors.forename: Marion
authors.initials: M
authors.lastname: Main
authors.forename: Luca
authors.initials: L
authors.lastname: Doglio
authors.forename: Luisa
authors.initials: L
authors.lastname: Politano
authors.forename: Filippo
authors.initials: F
authors.lastname: Cavallaro
authors.forename: Silvia
authors.initials: S
authors.lastname: Frosini
authors.forename: Luca
authors.initials: L
authors.lastname: Bello
authors.forename: Adelina
authors.initials: A
authors.lastname: Carlesi
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Bonetti
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Zucchini
authors.forename: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.forename: Marianna
authors.initials: M
authors.lastname: Scutifero
authors.forename: Flaviana
authors.initials: F
authors.lastname: Bianco
authors.forename: Francesca
authors.initials: F
authors.lastname: Rossi
authors.forename: Maria Chiara
authors.initials: MC
authors.lastname: Motta
authors.forename: Annalisa
authors.initials: A
authors.lastname: Sacco
authors.forename: Maria Alice
authors.initials: MA
authors.lastname: Donati
authors.forename: Tiziana
authors.initials: T
authors.lastname: Mongini
authors.forename: Antonella
authors.initials: A
authors.lastname: Pini
authors.forename: Roberta
authors.initials: R
authors.lastname: Battini
authors.forename: Elena
authors.initials: E
authors.lastname: Pegoraro
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Pasquini
authors.forename: Claudio
authors.initials: C
authors.lastname: Bruno
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Vita
authors.forename: Chiara
authors.initials: C
authors.lastname: de Waure
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D002648:Child; D005080:Exercise Test / Q000379:methods; D005684:Gait; D006801:Humans; D008297:Male; D018482:Muscle, Skeletal / Q000503:physiopathology*; D020388:Muscular Dystrophy, Duchenne / Q000503:physiopathology*; D011446:Prospective Studies; D016138:Walking