title: Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
abstract: The 6 minute walk test has been recently chosen as the primary outcome measure in international multicenter clinical trials in Duchenne muscular dystrophy ambulant patients. The aim of the study was to assess the spectrum of changes at 3 years in the individual measures, their correlation with steroid treatment, age and 6 minute walk test values at baseline. Ninety-six patients from 11 centers were assessed at baseline and 12, 24 and 36 months after baseline using the 6 minute walk test and the North Star Ambulatory Assessment. Three boys (3%) lost the ability to perform the 6 minute walk test within 12 months, another 13 between 12 and 24 months (14%) and 11 between 24 and 36 months (12%). The 6 minute walk test showed an average overall decline of -15.8 (SD 77.3) m at 12 months, of -58.9 (SD 125.7) m at 24 months and -104.22 (SD 146.2) m at 36 months. The changes were significantly different in the two baseline age groups and according to the baseline 6 minute walk test values (below and above 350 m) (p<0.001). The changes were also significantly different according to steroid treatment (p = 0.01). Similar findings were found for the North Star Ambulatory Assessment. These are the first 36 month longitudinal data using the 6 minute walk test and North Star Ambulatory Assessment in Duchenne muscular dystrophy. Our findings will help not only to have a better idea of the progression of the disorder but also provide reference data that can be used to compare with the results of the long term extension studies that are becoming available.
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: Elena Stacy
authors.initials: ES
authors.lastname: Mazzone
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Sivo
authors.affiliation: Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.
authors.forename: Maria Pia
authors.initials: MP
authors.lastname: Sormani
authors.affiliation: Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.affiliation: Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.affiliation: Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Adelina
authors.initials: A
authors.lastname: Carlesi
authors.affiliation: Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
authors.forename: Gianluca
authors.initials: G
authors.lastname: Vita
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Lavinia
authors.initials: L
authors.lastname: Fanelli
authors.affiliation: Child Neurology and Psychiatry Unit, "Casimiro Mondino" Foundation, Pavia, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Berardinelli
authors.affiliation: Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Yvan
authors.initials: Y
authors.lastname: Torrente
authors.affiliation: Neuromuscular Disease Unit, Giannina Gaslini Institute, Genoa, Italy.
authors.forename: Valentina
authors.initials: V
authors.lastname: Lanzillotta
authors.affiliation: Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy.
authors.forename: Emanuela
authors.initials: E
authors.lastname: Viggiano
authors.affiliation: Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy.
authors.forename: Paola
authors.initials: P
authors.lastname: D Ambrosio
authors.affiliation: Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
authors.forename: Filippo
authors.initials: F
authors.lastname: Cavallaro
authors.affiliation: Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Frosini
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Barp
authors.affiliation: Child Neurology and Psychiatry Unit, Maggiore Hospital, Bologna, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Bonfiglio
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: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: Neuromuscular Center, San Giovanni Battista Hospital, University of Turin, Turin, Italy.
authors.forename: Enrica
authors.initials: E
authors.lastname: Rolle
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Graziano
authors.affiliation: Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Magri
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Concetta
authors.initials: C
authors.lastname: Palermo
authors.affiliation: Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Rossi
authors.affiliation: Metabolic and Neuromuscular Unit, Meyer Hospital, Florence, Italy.
authors.forename: Maria Alice
authors.initials: MA
authors.lastname: Donati
authors.affiliation: Metabolic and Neuromuscular Unit, Meyer Hospital, Florence, Italy.
authors.forename: Michele
authors.initials: M
authors.lastname: Sacchini
authors.affiliation: Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy.
authors.forename: Maria Teresa
authors.initials: MT
authors.lastname: Arnoldi
authors.affiliation: Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Baranello
authors.affiliation: Neuromuscular Center, San Giovanni Battista Hospital, University of Turin, Turin, Italy.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Mongini
authors.affiliation: Child Neurology Unit, IRCCS Institute of Neurological Sciences, Bologna, Italy.
authors.forename: Antonella
authors.initials: A
authors.lastname: Pini
authors.affiliation: Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Battini
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Pegoraro
authors.affiliation: Department of Neurology, San Raffaele Scientific Institute, Milan, Italy.
authors.forename: Stefano
authors.initials: S
authors.lastname: Previtali
authors.affiliation: Neuromuscular Disease Unit, Giannina Gaslini Institute, Genoa, Italy.
authors.forename: Claudio
authors.initials: C
authors.lastname: Bruno
authors.affiliation: Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy.
authors.forename: Luisa
authors.initials: L
authors.lastname: Politano
authors.affiliation: Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
authors.forename: Giacomo P
authors.initials: GP
authors.lastname: Comi
authors.affiliation: Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D005080:Exercise Test; D006801:Humans; D007558:Italy; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000453:epidemiology*; D017063:Outcome Assessment, Health Care; D013997:Time Factors; D016138:Walking