title: 24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy.
abstract: OBJECTIVE
  The 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.
  
  
  METHODS
  One 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.
  
  
  RESULTS
  On 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.
  
  
  CONCLUSIONS
  These 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.
authors: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors: Elena Stacy
authors: ES
authors: Mazzone
authors: Marika
authors: M
authors: Pane
authors: Maria Pia
authors: MP
authors: Sormani
authors: Roberta
authors: R
authors: Scalise
authors: Angela
authors: A
authors: Berardinelli
authors: Sonia
authors: S
authors: Messina
authors: Yvan
authors: Y
authors: Torrente
authors: Adele
authors: A
authors: D'Amico
authors: Luca
authors: L
authors: Doglio
authors: Emanuela
authors: E
authors: Viggiano
authors: Paola
authors: P
authors: D'Ambrosio
authors: Filippo
authors: F
authors: Cavallaro
authors: Silvia
authors: S
authors: Frosini
authors: Luca
authors: L
authors: Bello
authors: Serena
authors: S
authors: Bonfiglio
authors: Roberto
authors: R
authors: De Sanctis
authors: Enrica
authors: E
authors: Rolle
authors: Flaviana
authors: F
authors: Bianco
authors: Francesca
authors: F
authors: Magri
authors: Francesca
authors: F
authors: Rossi
authors: Gessica
authors: G
authors: Vasco
authors: Gianluca
authors: G
authors: Vita
authors: Maria Chiara
authors: MC
authors: Motta
authors: Maria Alice
authors: MA
authors: Donati
authors: Michele
authors: M
authors: Sacchini
authors: Tiziana
authors: T
authors: Mongini
authors: Antonella
authors: A
authors: Pini
authors: Roberta
authors: R
authors: Battini
authors: Elena
authors: E
authors: Pegoraro
authors: Stefano
authors: S
authors: Previtali
authors: Sara
authors: S
authors: Napolitano
authors: Claudio
authors: C
authors: Bruno
authors: Luisa
authors: L
authors: Politano
authors: Giacomo Pietro
authors: GP
authors: Comi
authors: Enrico
authors: E
authors: Bertini
authors: Eugenio
authors: E
authors: Mercuri
mesh_terms: D000293:Adolescent; D000704:Analysis of Variance; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D005080:Exercise Test / Q000379:methods*; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D016015:Logistic Models; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000503:physiopathology*; D017063:Outcome Assessment, Health Care; D011241:Prednisone / Q000627:therapeutic use; D011282:Pregnenediones / Q000627:therapeutic use; D018570:Risk Assessment; D012307:Risk Factors; D013997:Time Factors; D016138:Walking / Q000502:physiology*
chemical_list: D000893:Anti-Inflammatory Agents; D005938:Glucocorticoids; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone