24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy

Elena Stacy Mazzone, Marika Pane, Maria Pia Sormani, Roberta Scalise, Angela Berardinelli, Sonia Messina, Yvan Torrente, Adele D'Amico, Luca Doglio, Emanuela Viggiano, Paola D'Ambrosio, Filippo Cavallaro, Silvia Frosini, Luca Bello, Serena Bonfiglio, Roberto de Sanctis, Enrica Rolle, Flaviana Bianco, Francesca Magri, Francesca RossiGessica Vasco, GianLuca L. Vita, Maria Chiara Motta, Maria Alice Donati, Michele Sacchini, Tiziana Mongini, Antonella Pini, Roberta Battini, Elena Pegoraro, Stefano Previtali, Sara Napolitano, Claudio Bruno, Luisa Politano, Giacomo Pietro Comi, Enrico Bertini, Eugenio Mercuri

Research output: Contribution to journalArticle

Abstract

Objectives: 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.

Original languageEnglish
Article numbere52512
JournalPLoS One
Volume8
Issue number1
DOIs
Publication statusPublished - Jan 17 2013

ASJC Scopus subject areas

  • Agricultural and Biological Sciences(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Medicine(all)

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    Mazzone, E. S., Pane, M., Sormani, M. P., 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., ... Mercuri, E. (2013). 24 Month Longitudinal Data in Ambulant Boys with Duchenne Muscular Dystrophy. PLoS One, 8(1), [e52512]. https://doi.org/10.1371/journal.pone.0052512