TY - JOUR
T1 - Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes
AU - Mercuri, Eugenio Maria
AU - Sormani, Maria Pia
AU - Messina, Sonia
AU - D'Amico, Adele
AU - Carlesi, Adelina
AU - Vita, Gianluca
AU - Berardinelli, Angela
AU - Torrente, Yvan
AU - Lanzillotta, Valentina
AU - Viggiano, Emanuela
AU - D'Ambrosio, Paola
AU - Cavallaro, Filippo
AU - Frosini, Silvia
AU - Barp, Andrea
AU - Bonfiglio, Serena
AU - Rolle, Enrica
AU - Magri, Francesca
AU - Rossi, Francesca
AU - Donati, Maria Alice
AU - Sacchini, Michele
AU - Arnoldi, Maria Teresa
AU - Baranello, Giovanni
AU - Mongini, Tiziana
AU - Pini, Antonella
AU - Battini, Roberta
AU - Pegoraro, Elena
AU - Previtali, Stefano
AU - Bruno, Claudio
AU - Politano, Luisa
AU - Comi, Giacomo P.
AU - Bertini, Enrico
PY - 2014
Y1 - 2014
N2 - 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.
AB - 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.
KW - Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes
KW - Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes
UR - http://hdl.handle.net/10807/65130
U2 - 10.1371/journal.pone.0108205
DO - 10.1371/journal.pone.0108205
M3 - Article
VL - 9
SP - e108205-e108205
JO - PLoS One
JF - PLoS One
SN - 1932-6203
ER -