PLoS ONE (2014-01-01)

Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes.

  • Marika Pane,
  • Elena Stacy Mazzone,
  • Serena Sivo,
  • Maria Pia Sormani,
  • Sonia Messina,
  • Adele D'Amico,
  • Adelina Carlesi,
  • Gianluca Vita,
  • Lavinia Fanelli,
  • Angela Berardinelli,
  • Yvan Torrente,
  • Valentina Lanzillotta,
  • Emanuela Viggiano,
  • Paola D Ambrosio,
  • Filippo Cavallaro,
  • Silvia Frosini,
  • Andrea Barp,
  • Serena Bonfiglio,
  • Roberta Scalise,
  • Roberto De Sanctis,
  • Enrica Rolle,
  • Alessandra Graziano,
  • Francesca Magri,
  • Concetta Palermo,
  • Francesca Rossi,
  • Maria Alice Donati,
  • Michele Sacchini,
  • Maria Teresa Arnoldi,
  • Giovanni Baranello,
  • Tiziana Mongini,
  • Antonella Pini,
  • Roberta Battini,
  • Elena Pegoraro,
  • Stefano Previtali,
  • Claudio Bruno,
  • Luisa Politano,
  • Giacomo P Comi,
  • Enrico Bertini,
  • Eugenio Mercuri

DOI
https://doi.org/10.1371/journal.pone.0108205
Journal volume & issue
Vol. 9, no. 10
p. e108205

Abstract

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The 6 minute walk test has been recently chosen as the primary outcome measure in international multicenter clinical trials in Duchenne muscular dystrophy ambulant patients. The aim of the study was to assess the spectrum of changes at 3 years in the individual measures, their correlation with steroid treatment, age and 6 minute walk test values at baseline. Ninety-six patients from 11 centers were assessed at baseline and 12, 24 and 36 months after baseline using the 6 minute walk test and the North Star Ambulatory Assessment. Three boys (3%) lost the ability to perform the 6 minute walk test within 12 months, another 13 between 12 and 24 months (14%) and 11 between 24 and 36 months (12%). The 6 minute walk test showed an average overall decline of -15.8 (SD 77.3) m at 12 months, of -58.9 (SD 125.7) m at 24 months and -104.22 (SD 146.2) m at 36 months. The changes were significantly different in the two baseline age groups and according to the baseline 6 minute walk test values (below and above 350 m) (p<0.001). The changes were also significantly different according to steroid treatment (p = 0.01). Similar findings were found for the North Star Ambulatory Assessment. These are the first 36 month longitudinal data using the 6 minute walk test and North Star Ambulatory Assessment in Duchenne muscular dystrophy. Our findings will help not only to have a better idea of the progression of the disorder but also provide reference data that can be used to compare with the results of the long term extension studies that are becoming available.