• Plos One · Jan 2013

    Multicenter Study

    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 Rossi, Gessica Vasco, Gianluca 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, and Eugenio Mercuri.
    • Department of Paediatric Neurology, Catholic University, Rome, Italy.
    • Plos One. 2013 Jan 1; 8 (1): e52512.

    ObjectivesThe 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.MethodsOne 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.ResultsOn 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.ConclusionsThese 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.

      Pubmed     Free full text   Copy Citation     Plaintext  

      Add institutional full text...

    Notes

     
    Knowledge, pearl, summary or comment to share?
    300 characters remaining
    help        
    You can also include formatting, links, images and footnotes in your notes
    • Simple formatting can be added to notes, such as *italics*, _underline_ or **bold**.
    • Superscript can be denoted by <sup>text</sup> and subscript <sub>text</sub>.
    • Numbered or bulleted lists can be created using either numbered lines 1. 2. 3., hyphens - or asterisks *.
    • Links can be included with: [my link to pubmed](http://pubmed.com)
    • Images can be included with: ![alt text](https://bestmedicaljournal.com/study_graph.jpg "Image Title Text")
    • For footnotes use [^1](This is a footnote.) inline.
    • Or use an inline reference [^1] to refer to a longer footnote elseweher in the document [^1]: This is a long footnote..

    hide…