title: Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study.
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.
authors: Department of Paediatric Neurology, Catholic University, Rome.
authors: E
authors: E
authors: Mazzone
authors: G
authors: G
authors: Vasco
authors: M P
authors: MP
authors: Sormani
authors: Y
authors: Y
authors: Torrente
authors: A
authors: A
authors: Berardinelli
authors: S
authors: S
authors: Messina
authors: A
authors: A
authors: D'Amico
authors: L
authors: L
authors: Doglio
authors: L
authors: L
authors: Politano
authors: F
authors: F
authors: Cavallaro
authors: S
authors: S
authors: Frosini
authors: L
authors: L
authors: Bello
authors: S
authors: S
authors: Bonfiglio
authors: E
authors: E
authors: Zucchini
authors: R
authors: R
authors: De Sanctis
authors: M
authors: M
authors: Scutifero
authors: F
authors: F
authors: Bianco
authors: F
authors: F
authors: Rossi
authors: M C
authors: MC
authors: Motta
authors: A
authors: A
authors: Sacco
authors: M A
authors: MA
authors: Donati
authors: T
authors: T
authors: Mongini
authors: A
authors: A
authors: Pini
authors: R
authors: R
authors: Battini
authors: E
authors: E
authors: Pegoraro
authors: M
authors: M
authors: Pane
authors: S
authors: S
authors: Gasperini
authors: S
authors: S
authors: Previtali
authors: S
authors: S
authors: Napolitano
authors: D
authors: D
authors: Martinelli
authors: C
authors: C
authors: Bruno
authors: G
authors: G
authors: Vita
authors: G
authors: G
authors: Comi
authors: E
authors: E
authors: Bertini
authors: E
authors: E
authors: Mercuri
mesh_terms: D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003430:Cross-Sectional Studies; D018450:Disease Progression; D005260:Female; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000503:physiopathology*; D011239:Prednisolone / Q000627:therapeutic use; D011282:Pregnenediones / Q000627:therapeutic use; D015203:Reproducibility of Results; D012720:Severity of Illness Index; D013223:Statistics as Topic; D016138:Walking / Q000502:physiology
chemical_list: D000893:Anti-Inflammatory Agents; D011282:Pregnenediones; D011239:Prednisolone; C021988:deflazacort