North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy.
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.
Mazzone
Elena
E
Department of Paediatric Neurology, Catholic University, Rome, Italy.
Martinelli
Diego
D
Berardinelli
Angela
A
Messina
Sonia
S
D'Amico
Adele
A
Vasco
Gessica
G
Main
Marion
M
Doglio
Luca
L
Politano
Luisa
L
Cavallaro
Filippo
F
Frosini
Silvia
S
Bello
Luca
L
Carlesi
Adelina
A
Bonetti
Anna Maria
AM
Zucchini
Elisabetta
E
De Sanctis
Roberto
R
Scutifero
Marianna
M
Bianco
Flaviana
F
Rossi
Francesca
F
Motta
Maria Chiara
MC
Sacco
Annalisa
A
Donati
Maria Alice
MA
Mongini
Tiziana
T
Pini
Antonella
A
Battini
Roberta
R
Pegoraro
Elena
E
Pane
Marika
M
Pasquini
Elisabetta
E
Bruno
Claudio
C
Vita
Giuseppe
G
de Waure
Chiara
C
Bertini
Enrico
E
Mercuri
Eugenio
E
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