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

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 stero...

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Bibliographic Details
Published in:PloS one 2014-10, Vol.9 (10), p.e108205-e108205
Main Authors: Pane, Marika, Mazzone, Elena Stacy, Sivo, Serena, Sormani, Maria Pia, Messina, Sonia, D'Amico, Adele, Carlesi, Adelina, Vita, Gianluca, Fanelli, Lavinia, Berardinelli, Angela, Torrente, Yvan, Lanzillotta, Valentina, Viggiano, Emanuela, D Ambrosio, Paola, Cavallaro, Filippo, Frosini, Silvia, Barp, Andrea, Bonfiglio, Serena, Scalise, Roberta, De Sanctis, Roberto, Rolle, Enrica, Graziano, Alessandra, Magri, Francesca, Palermo, Concetta, Rossi, Francesca, Donati, Maria Alice, Sacchini, Michele, Arnoldi, Maria Teresa, Baranello, Giovanni, Mongini, Tiziana, Pini, Antonella, Battini, Roberta, Pegoraro, Elena, Previtali, Stefano, Bruno, Claudio, Politano, Luisa, Comi, Giacomo P, Bertini, Enrico, Mercuri, Eugenio
Format: Article
Language:English
Subjects:
Anesthesiology
Biology and Life Sciences
Child
Child, Preschool
Children & youth
Clinical trials
Correlation analysis
Disease Progression
Duchenne's muscular dystrophy
Dystrophy
Exercise Test
Humans
Italy
Longitudinal Studies
Male
Medical research
Medicine and Health Sciences
Metabolism
Muscular dystrophy
Muscular Dystrophy, Duchenne - epidemiology
Neurology
Neuromuscular diseases
Neurosciences
Observational studies
Outcome Assessment, Health Care
Patients
Psychiatry
Steroids
Studies
Time Factors
Walking
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Summary: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
ISSN:1932-6203
1932-6203
DOI:10.1371/journal.pone.0108205