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Quantitative muscle MRI: A powerful surrogate outcome measure in Duchenne muscular dystrophy

Bonati, Ulrike and Hafner, Patricia and Schadelin, Sabine and Schmid, Maurice and Naduvilekoot Devasia, Arjith and Schroeder, Jonas and Zuesli, Stephanie and Pohlman, Urs and Neuhaus, Cornelia and Klein, Andrea and Sinnreich, Michael and Haas, Tanja and Gloor, Monika and Bieri, Oliver and Fischmann, Arne and Fischer, Dirk. (2015) Quantitative muscle MRI: A powerful surrogate outcome measure in Duchenne muscular dystrophy. Neuromuscular Disorders, 25 (9). pp. 679-685.

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Official URL: https://edoc.unibas.ch/62456/

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Abstract

In muscular dystrophies quantitative muscle MRI (qMRI) detects disease progression more sensitively than clinical scores. This prospective one year observational study compared qMRI with clinical scores in Duchenne muscular dystrophy (DMD) to investigate if qMRI can serve as a surrogate outcome measure in clinical trials. In 20 DMD patients the motor function measure (MFM) total and subscores (D1-D3) were done for physical examination, and the fat fraction (MFF) of thigh muscle qMRI was obtained using the two-point Dixon method. Effect sizes (ES) were calculated for all measures. Sample size estimation (SS) was done modelling assumed treatment effects. Ambulant patients 7 years (highest ES in the MFM D1 subscore (1.2)), and non-ambulant patients (highest ES in the total MFM score (0.7)) worsened. In comparison the ES of QMRI was much larger, e.g. SS estimations for qMRI data were up to 17 fold smaller compared to the MFM total score and up to 7 fold to the D1 subscore, respectively. QMRI shows pathophysiological changes in DMD and might serve as a surrogate outcome measure in clinical trials.
Faculties and Departments:03 Faculty of Medicine > Departement Biomedizin > Department of Biomedicine, University Hospital Basel > Neuromuscular Research (Sinnreich)
UniBasel Contributors:Sinnreich, Michael
Item Type:Article, refereed
Article Subtype:Research Article
ISSN:1873-2364 (Electronic) 0960-8966 (Linking)
Note:Publication type according to Uni Basel Research Database: Journal article
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Last Modified:24 Nov 2018 14:03
Deposited On:24 Nov 2018 14:03

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