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PLoS One. 2014 Oct 1;9(10):e108205. doi: 10.1371/journal.pone.0108205. eCollection 2014.

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.

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Department of Paediatric Neurology, Catholic University, Rome, Italy.
Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.
Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy.
Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
Child Neurology and Psychiatry Unit, "Casimiro Mondino" Foundation, Pavia, Italy.
Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Neuromuscular Disease Unit, Giannina Gaslini Institute, Genoa, Italy.
Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy.
Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy.
Department of Neurosciences, University of Padua, Padua, Italy.
Child Neurology and Psychiatry Unit, Maggiore Hospital, Bologna, Italy.
Neuromuscular Center, San Giovanni Battista Hospital, University of Turin, Turin, Italy.
Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy.
Metabolic and Neuromuscular Unit, Meyer Hospital, Florence, Italy.
Child Neurology Unit, IRCCS Institute of Neurological Sciences, Bologna, Italy.
Department of Neurology, San Raffaele Scientific Institute, Milan, Italy.


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.

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