Functional changes in Duchenne muscular dystrophy: A 12-month longitudinal cohort study
| Autor: | Luisa Politano, Silvia Frosini, MP Sormani, Tiziana Mongini, Enrico Bertini, Adele D'Amico, Angela Berardinelli, Sara Napolitano, Roberta Battini, Eugenio Mercuri, Giacomo P. Comi, Maria Chiara Motta, Maria Benedetta Donati, Stefano C. Previtali, Marika Pane, Serena Gasperini, Flaviana Bianco, Fabio Cavallaro, Luca Doglio, Gianluca Vita, Sonia Messina, Elena Pegoraro, Danilo Martinelli, Angela Sacco, Gessica Vasco, M Scutifero, Luca Bello, Yvan Torrente, Francesca Rossi, E. Zucchini, Cristina Bruno, Elena S. Mazzone, Antonella Pini, R. De Sanctis, S Bonfiglio |
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| Přispěvatelé: | Mazzone, E, Vasco, G, Sormani, Mp, Torrente, Y, Berardinelli, A, Messina, S, D’Amico, A, Doglio, L, Politano, L, Cavallaro, F, Frosini, S, Bello, L, Bonfiglio, S, Zucchini, E, De Sanctis, R, Scutifero, M, Bianco, F, Rossi, F, Motta, Mc, Sacco, A, Donati, Ma, Mongini, T, Pini, A, Battini, R, Pegoraro, E, Pane, M, Gasperini, S, Previtali, S, Napolitano, S, Martinelli, D, Bruno, C, Vita, G, Comi, Giancarlo, Bertini, E, Mercuri, E. |
| Rok vydání: | 2011 |
| Předmět: |
Male
medicine.medical_specialty Pediatrics Adolescent Cross-sectional study Prednisolone Duchenne muscular dystrophy Statistics as Topic Anti-Inflammatory Agents Walking Severity of Illness Index Cohort Studies Settore MED/39 - NEUROPSICHIATRIA INFANTILE Pregnenediones Severity of illness medicine Humans Muscular Dystrophy Muscular dystrophy Child Preschool business.industry Reproducibility of Results drug therapy/physiopathology Duchenne medicine.disease Muscular Dystrophy Duchenne Clinical trial Cross-Sectional Studies therapeutic use Child Preschool physiology Ambulatory Cohort Disease Progression Physical therapy Female Neurology (clinical) business Adolescent Anti-Inflammatory Agents therapeutic use Child Child Preschool Cohort Studies Cross-Sectional Studies Disease Progression Female Humans Male Muscular Dystrophy drug therapy/physiopathology Prednisolone therapeutic use Pregnenediones therapeutic use Reproducibility of Results Severity of Illness Index Statistics as Topic Walking Cohort study |
| Zdroj: | ResearcherID |
| ISSN: | 1526-632X 0028-3878 |
| DOI: | 10.1212/wnl.0b013e318225ab2e |
| Popis: | Objective: The aim of the study was to assess different outcome measures in a cohort of ambulant boys with Duchenne muscular dystrophy (DMD) over 12 months in order to establish the spectrum of possible changes in relation to age and steroid treatment. Methods: The study is a longitudinal multicentric cohort study. A total of 106 ambulant patients with DMD were assessed using the 6-minute walk test (6MWT) and North Star Ambulatory Assessment (NSAA) at baseline and 12 months. Clinical data including age and steroid treatment were collected. Results: During the 12 months of the study, we observed a mean decline of 25.8 meters in the 6MWT with a SD of 74.3 meters. On NSAA, the mean decline was 2.2 points with a SD of 3.7. Not all the boys with DMD in our cohort showed a decline over the 12 months, with young boys showing some improvement in their 6MWT and NSAA scores up to the age of 7. NSAA and the 6MWT had the highest correlation ( r = 0.52, p Conclusions: This study provides longitudinal data of NSAA and 6MWT over a 12-month period. These data can be useful when designing a clinical trial. |
| Databáze: | OpenAIRE |
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