Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study

Accedi al documento(opens in a new window)|View at Publisher| Export | Download | Add to List | More... Neurology Volume 77, Issue 3, 19 July 2011, Pages 250-256 Open Access Functional changes in Duchenne muscular dystrophy (Article) Mazzone, E.a, Vasco, G.a, Sormani, M.P.abe, Torrente, Y.c, Berardinelli, A.d, Messina, S.i, D'Amico, A.f, Doglio, L.h, Politano, L.i, Cavallaro, F.e, Frosini, S.j, Bello, L.l, Bonfiglio, S.c, Zucchini, E.m, De Sanctis, R.a, Scutifero, M.a, Bianco, F.a, Rossi, F.n, Motta, M.C.d, Sacco, A.a, Donati, M.A.n, Mongini, T.n, Pini, A.m, Battini, R.k, Pegoraro, E.l, Pane, M.a, Gasperini, S.n, Previtali, S.o, Napolitano S.o, Martinelli, D.ag, Bruno, C.hk, Vita, G.e, Comi, G.c, Bertini, E.f, Mercuri, E.ap a Department of Paediatric Neurology, Catholic University, Rome, Italy b Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy c Department of Neurological Sciences, IRCCS Ospedale Maggiore Policlinico, University of Milan, Milan, Italy View additional affiliations View references (15) Abstract 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 > 0.001). 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.