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

Neuromuscular Disorders

Volumn 25, Issue 9, 2015, Pages 679-685

  Bonati, Ulrike ^a,b   Hafner, Patricia ^b   Schädelin, Sabine ^b   Schmid, Maurice ^a   Naduvilekoot Devasia, Arjith ^a   Schroeder, Jonas ^a   Zuesli, Stephanie ^a   Pohlman, Urs ^a   Neuhaus, Cornelia ^a   Klein, Andrea ^c   Sinnreich, Michael ^b   Haas, Tanja ^b   Gloor, Monika ^b   Bieri, Oliver ^b   Fischmann, Arne ^b   Fischer, Dirk ^a,b  

^a UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^b UNIVERSITY HOSPITAL BASEL   (Switzerland)

^c UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

Author keywords

Duchenne muscular dystrophy; Endpoint; Neuromuscular disorders; Outcome measure; Quantitative MRI

Indexed keywords

ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL ARTICLE; COMPARATIVE STUDY; CONTROLLED STUDY; CORRELATION ANALYSIS; CROSS-SECTIONAL STUDY; D1 SCORE; DUCHENNE MUSCULAR DYSTROPHY; EFFECT SIZE; HUMAN; INTERMETHOD COMPARISON; LONGITUDINAL STUDY; MOBILIZATION; MOTOR FAT FRACTION; MOTOR FUNCTION MEASURE; MYOPATHY; NEUROLOGIC EXAMINATION; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SCANNER; OBSERVATIONAL STUDY; OUTCOME ASSESSMENT; PATHOPHYSIOLOGY; PHYSICAL EXAMINATION; PRESCHOOL CHILD; PRIORITY JOURNAL; QUANTITATIVE ANALYSIS; RATING SCALE; SAMPLE SIZE; SCHOOL CHILD; SCORING SYSTEM; SENSITIVITY ANALYSIS; THERAPY EFFECT; DISEASE COURSE; FOLLOW UP; MALE; PATHOLOGY; PROCEDURES; PROSPECTIVE STUDY; SKELETAL MUSCLE;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; DISEASE PROGRESSION; FOLLOW-UP STUDIES; HUMANS; MAGNETIC RESONANCE IMAGING; MALE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; PROSPECTIVE STUDIES;

EID: 84939571490     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2015.05.006     Document Type: Article

References (25)

1. Mazzone E., Vasco G., Sormani M.P., et al. Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study. Neurology 2011, 77:250-256.
2. Vuillerot C., Girardot F., Payan C., et al. Monitoring changes and predicting loss of ambulation in Duchenne muscular dystrophy with the Motor Function Measure. Dev Med Child Neurol 2010, 52:60-65.
3. Sinclair C.D.J., Morrow J.M., Yousry T.A., et al. Inter-scan reproducibility of quantitative neuromuscular MRI. Neuromuscul Disord 2010, 20:S28.
4. Fischmann A., Gloor M., Fasler S., et al. Muscular involvement assessed by MRI correlates to motor function measurement values in oculopharyngeal muscular dystrophy. J Neurol 2011, 258:1333-1340.
5. Fischmann A., Hafner P., Fasler S., et al. Quantitative MRI can detect subclinical disease progression in muscular dystrophy. J Neurol 2012, 259:1648-1654.
6. Wokke B.H., Bos C., Reijnierse M., et al. Comparison of Dixon and T1-weighted MR methods to assess the degree of fat infiltration in Duchenne muscular dystrophy patients. J Magn Reson Imaging 2013, 38:619-624.
7. Ponrartana S., Ramos-Platt L., Wren T.A., et al. Effectiveness of diffusion tensor imaging in assessing disease severity in Duchenne muscular dystrophy: preliminary study. Pediatr Radiol 2015, 45:582-589.
8. Forbes S.C., Willcocks R.J., Triplett W.T., et al. Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional study. PLoS ONE 2014, 9:e106435.
9. Wren T.A., Bluml S., Tseng-Ong L., Gilsanz V. Three-point technique of fat quantification of muscle tissue as a marker of disease progression in Duchenne muscular dystrophy: preliminary study. AJR Am J Roentgenol 2008, 190:W8-12.
10. Arpan I., Willcocks R.J., Forbes S.C., et al. Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS. Neurology 2014, 83:974-980.
11. Pichiecchio A., Uggetti C., Egitto M.G., et al. Quantitative MR evaluation of body composition in patients with Duchenne muscular dystrophy. Eur Radiol 2002, 12:2704-2709.
12. Garrood P., Hollingsworth K.G., Eagle M., et al. MR imaging in Duchenne muscular dystrophy: quantification of T1-weighted signal, contrast uptake, and the effects of exercise. J Magn Reson Imaging 2009, 30:1130-1138.
13. Huang Y., Majumdar S., Genant H.K., et al. Quantitative MR relaxometry study of muscle composition and function in Duchenne muscular dystrophy. J Magn Reson Imaging 1994, 4:59-64.
14. Fischmann A., Hafner P., Gloor M., et al. Quantitative MRI and loss of free ambulation in Duchenne muscular dystrophy. J Neurol 2013, 260:969-974.
15. Willcocks R.J., Arpan I.A., Forbes S.C., et al. Longitudinal measurements of MRI-T2 in boys with Duchenne muscular dystrophy: effects of age and disease progression. Neuromuscul Disord 2014, 24:393-401.
16. Gaeta M., Messina S., Mileto A., et al. Muscle fat-fraction and mapping in Duchenne muscular dystrophy: evaluation of disease distribution and correlation with clinical assessments. Preliminary experience. Skeletal Radiol 2012, 41:955-961.
17. European Medicines Agency Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy (draft) EMA/CHMP/236981/2011. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/03/WC500139508.pdf.
18. Fischmann A., Morrow J.M., Sinclair C.D., et al. Improved anatomical reproducibility in quantitative lower-limb muscle MRI. J Magn Reson Imaging 2014, 39:1033-1038.
19. Skinner T.E., Glover G.H. An extended two-point Dixon algorithm for calculating separate water, fat, and B0 images. Magn Reson Med 1997, 37:628-630.
20. McDonald C.M., Henricson E.K., Han J.J., et al. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve 2010, 41:500-510.
21. Mazzone E.S., Messina S., Vasco G., et al. Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscul Disord 2009, 19:458-461.
22. Arpan I., Forbes S.C., Lott D.J., et al. T(2) mapping provides multiple approaches for the characterization of muscle involvement in neuromuscular diseases: a cross-sectional study of lower leg muscles in 5-15-year-old boys with Duchenne muscular dystrophy. NMR Biomed 2013, 26:320-328.
23. Fleming T.R., Powers J.H. Biomarkers and surrogate endpoints in clinical trials. Stat Med 2012, 31:2973-2984.
24. Wokke B.H., van den Bergen J.C., Versluis M.J., et al. Quantitative MRI and strength measurements in the assessment of muscle quality in Duchenne muscular dystrophy. Neuromuscul Disord 2014, 24:409-416.
25. McDonald C.M., Henricson E.K., Abresch R.T., et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve 2013, 48:343-356.