24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy
| العنوان: | 24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy |
|---|---|
| المؤلفون: | Claudio Bruno, Enrico Bertini, Silvia Frosini, Gianluca Vita, Flaviana Bianco, Luisa Politano, Sara Napolitano, Maria Pia Sormani, Adele D'Amico, Stefano C. Previtali, Marika Pane, Angela Berardinelli, Emanuela Viggiano, Francesca Rossi, Filippo Cavallaro, Sonia Messina, Elena Pegoraro, Tiziana Mongini, Yvan Torrente, Antonella Pini, Roberto De Sanctis, Gessica Vasco, Eugenio Mercuri, Elena S. Mazzone, Francesca Magri, Enrica Rolle, Roberta Scalise, Paola D'Ambrosio, Roberta Battini, Giacomo P. Comi, Luca Bello, Maria Chiara Motta, Maria Alice Donati, Luca Doglio, Serena Bonfiglio, Michele Sacchini |
| المساهمون: | Mazzone, E, Pane, M, Sormani, Mp, Scalise, R, Berardinelli, A, Messina, S, Torrente, Y, D'Amico, A, Doglio, L, Viggiano, E, D'Ambrosio, P, Cavallaro, F, Frosini, S, Bello, L, Bonfiglio, S, De Sanctis, R, Rolle, E, Bianco, F, Magri, F, Rossi, F, Vasco, G, Vita, G, Motta, Mc, Donati, Ma, Sacchini, M, Mongini, T, Pini, A, Battini, R, Pegoraro, E, Previtali, S, Napolitano, S, Bruno, C, Politano, Luisa, Comi, Gp, Bertini, E, Mercuri, E. |
| المصدر: | PLoS ONE PLoS ONE; Vol 8 PLoS ONE, Vol 8, Iss 1, p e52512 (2013) |
| سنة النشر: | 2013 |
| مصطلحات موضوعية: | Male, Time Factors, Outcome Assessment, Non-Clinical Medicine, Duchenne muscular dystrophy, Anti-Inflammatory Agents, Duchenne Muscular Dystrophy, Walking, Outcome assessment, Cohort Studies, 0302 clinical medicine, Pregnenediones, Risk Factors, Outcome Assessment, Health Care, Medicine, Muscular Dystrophy, Longitudinal Studies, Muscular dystrophy, 10. No inequality, Child, 0303 health sciences, Multidisciplinary, Statistics, 3. Good health, Child, Preschool, Ambulatory, outcome, Risk assessment, Research Article, Test Evaluation, medicine.medical_specialty, Adolescent, Longitudinal data, Clinical Research Design, Science, Biostatistics, Natural history of disease, Risk Assessment, 03 medical and health sciences, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Diagnostic Medicine, Humans, 6-minute walk test, Clinical Trials, Analysis of Variance, Exercise Test, Glucocorticoids, Logistic Models, Muscular Dystrophy, Duchenne, Prednisone, Preschool, 030304 developmental biology, Clinical Genetics, Health Care Policy, business.industry, Health Risk Analysis, X-Linked, Duchenne, medicine.disease, Health Care, Physical therapy, business, 030217 neurology & neurosurgery, Mathematics |
| الوصف: | ObjectivesThe aim of the study was i) to assess the spectrum of changes over 24 months in ambulant boys affected by Duchenne muscular dystrophy, ii) to establish the difference between the first and the second year results and iii) to identify possible early markers of loss of ambulation.MethodsOne hundred and thirteen patients (age range 4.1-17, mean 8.2) fulfilled the inclusion criteria, 67 of the 113 were on daily and 40 on intermittent steroids, while 6 were not on steroids. All were assessed using the 6 Minute Walk Test (6MWT), the North Star Ambulatory Assessment (NSAA) and timed test.ResultsOn the 6MWT there was an average overall decline of -22.7 (SD 81.0) in the first year and of -64.7 (SD 123.1) in the second year. On the NSAA the average overall decline was of -1.86 (SD 4.21) in the first year and of -2.98 (SD 5.19) in the second year. Fourteen children lost ambulation, one in the first year and the other 13 in the second year of the study. A distance of at least 330 meters on the 6MWT, or a NSAA score of 18 at baseline reduced significantly the risk of losing ambulation within 2 years.ConclusionsThese results can be of help at the time of using inclusion criteria for a study in ambulant patients in order to minimize the risk of patients who may lose ambulation within the time of the trial. |
| اللغة: | English |
| تدمد: | 1932-6203 |
| DOI: | 10.1371/journal.pone.0052512 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cd64f760c0c89949a47f5b160131bea1 |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....cd64f760c0c89949a47f5b160131bea1 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 19326203 |
|---|---|
| DOI: | 10.1371/journal.pone.0052512 |