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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Autores principales:	Marika Pane, Elena Stacy Mazzone, Serena Sivo, Maria Pia Sormani, Sonia Messina, Adele D'Amico, Adelina Carlesi, Gianluca Vita, Lavinia Fanelli, Angela Berardinelli, Yvan Torrente, Valentina Lanzillotta, Emanuela Viggiano, Paola D Ambrosio, Filippo Cavallaro, Silvia Frosini, Andrea Barp, Serena Bonfiglio, Roberta Scalise, Roberto De Sanctis, Enrica Rolle, Alessandra Graziano, Francesca Magri, Concetta Palermo, Francesca Rossi, Maria Alice Donati, Michele Sacchini, Maria Teresa Arnoldi, Giovanni Baranello, Tiziana Mongini, Antonella Pini, Roberta Battini, Elena Pegoraro, Stefano Previtali, Claudio Bruno, Luisa Politano, Giacomo P Comi, Enrico Bertini, Eugenio Mercuri
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2014
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/648f7174ca734920ac93ed77179799f2
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spelling	oai:doaj.org-article:648f7174ca734920ac93ed77179799f22021-11-25T05:58:21ZLong term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.1932-620310.1371/journal.pone.0108205https://doaj.org/article/648f7174ca734920ac93ed77179799f22014-01-01T00:00:00Zhttps://doi.org/10.1371/journal.pone.0108205https://doaj.org/toc/1932-6203The 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.Marika PaneElena Stacy MazzoneSerena SivoMaria Pia SormaniSonia MessinaAdele D'AmicoAdelina CarlesiGianluca VitaLavinia FanelliAngela BerardinelliYvan TorrenteValentina LanzillottaEmanuela ViggianoPaola D AmbrosioFilippo CavallaroSilvia FrosiniAndrea BarpSerena BonfiglioRoberta ScaliseRoberto De SanctisEnrica RolleAlessandra GrazianoFrancesca MagriConcetta PalermoFrancesca RossiMaria Alice DonatiMichele SacchiniMaria Teresa ArnoldiGiovanni BaranelloTiziana MonginiAntonella PiniRoberta BattiniElena PegoraroStefano PrevitaliClaudio BrunoLuisa PolitanoGiacomo P ComiEnrico BertiniEugenio MercuriPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 9, Iss 10, p e108205 (2014)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Marika Pane Elena Stacy Mazzone Serena Sivo Maria Pia Sormani Sonia Messina Adele D'Amico Adelina Carlesi Gianluca Vita Lavinia Fanelli Angela Berardinelli Yvan Torrente Valentina Lanzillotta Emanuela Viggiano Paola D Ambrosio Filippo Cavallaro Silvia Frosini Andrea Barp Serena Bonfiglio Roberta Scalise Roberto De Sanctis Enrica Rolle Alessandra Graziano Francesca Magri Concetta Palermo Francesca Rossi Maria Alice Donati Michele Sacchini Maria Teresa Arnoldi Giovanni Baranello Tiziana Mongini Antonella Pini Roberta Battini Elena Pegoraro Stefano Previtali Claudio Bruno Luisa Politano Giacomo P Comi Enrico Bertini Eugenio Mercuri Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
description	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.
format	article
author	Marika Pane Elena Stacy Mazzone Serena Sivo Maria Pia Sormani Sonia Messina Adele D'Amico Adelina Carlesi Gianluca Vita Lavinia Fanelli Angela Berardinelli Yvan Torrente Valentina Lanzillotta Emanuela Viggiano Paola D Ambrosio Filippo Cavallaro Silvia Frosini Andrea Barp Serena Bonfiglio Roberta Scalise Roberto De Sanctis Enrica Rolle Alessandra Graziano Francesca Magri Concetta Palermo Francesca Rossi Maria Alice Donati Michele Sacchini Maria Teresa Arnoldi Giovanni Baranello Tiziana Mongini Antonella Pini Roberta Battini Elena Pegoraro Stefano Previtali Claudio Bruno Luisa Politano Giacomo P Comi Enrico Bertini Eugenio Mercuri
author_facet	Marika Pane Elena Stacy Mazzone Serena Sivo Maria Pia Sormani Sonia Messina Adele D'Amico Adelina Carlesi Gianluca Vita Lavinia Fanelli Angela Berardinelli Yvan Torrente Valentina Lanzillotta Emanuela Viggiano Paola D Ambrosio Filippo Cavallaro Silvia Frosini Andrea Barp Serena Bonfiglio Roberta Scalise Roberto De Sanctis Enrica Rolle Alessandra Graziano Francesca Magri Concetta Palermo Francesca Rossi Maria Alice Donati Michele Sacchini Maria Teresa Arnoldi Giovanni Baranello Tiziana Mongini Antonella Pini Roberta Battini Elena Pegoraro Stefano Previtali Claudio Bruno Luisa Politano Giacomo P Comi Enrico Bertini Eugenio Mercuri
author_sort	Marika Pane
title	Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
title_short	Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
title_full	Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
title_fullStr	Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
title_full_unstemmed	Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.
title_sort	long term natural history data in ambulant boys with duchenne muscular dystrophy: 36-month changes.
publisher	Public Library of Science (PLoS)
publishDate	2014
url	https://doaj.org/article/648f7174ca734920ac93ed77179799f2
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Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.

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