Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis.

Details

Serval ID
serval:BIB_867D1F86028F
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis.
Journal
Neuromuscular disorders
Author(s)
Schmidt S., Hafner P., Klein A., Rubino-Nacht D., Gocheva V., Schroeder J., Naduvilekoot Devasia A., Zuesli S., Bernert G., Laugel V., Bloetzer C., Steinlin M., Capone A., Gloor M., Tobler P., Haas T., Bieri O., Zumbrunn T., Fischer D., Bonati U.
ISSN
1873-2364 (Electronic)
ISSN-L
0960-8966
Publication state
Published
Issued date
01/2018
Peer-reviewed
Oui
Volume
28
Number
1
Pages
16-23
Language
english
Notes
Publication types: Journal Article
Publication Status: ppublish
Abstract
The development of new therapeutic agents for the treatment of Duchenne muscular dystrophy has put a focus on defining outcome measures most sensitive to capture treatment effects. This cross-sectional analysis investigates the relation between validated clinical assessments such as the 6-minute walk test, motor function measure and quantitative muscle MRI of thigh muscles in ambulant Duchenne muscular dystrophy patients, aged 6.5 to 10.8 years (mean 8.2, SD 1.1). Quantitative muscle MRI included the mean fat fraction using a 2-point Dixon technique, and transverse relaxation time (T2) measurements. All clinical assessments were highly significantly inter-correlated with p < 0.001. The strongest correlation with the motor function measure and its D1-subscore was shown by the 6-minute walk test. Clinical assessments showed no correlation with age. Importantly, quantitative muscle MRI values significantly correlated with all clinical assessments with the extensors showing the strongest correlation. In contrast to the clinical assessments, quantitative muscle MRI values were highly significantly correlated with age. In conclusion, the motor function measure and timed function tests measure disease severity in a highly comparable fashion and all tests correlated with quantitative muscle MRI values quantifying fatty muscle degeneration.

Keywords
6-minute walk distance, 6-minute walk test, Duchenne muscular dystrophy, Imaging biomarkers, Motor function measure, Quantitative MRI, Timed function tests
Pubmed
Web of science
Create date
30/11/2017 19:16
Last modification date
20/08/2019 14:45
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