P1.41 Design of a multi-center study to examine skeletal muscles of children with Duchenne muscular dystrophy using MRI/MRS

Forbes, Sean C.; Walter, Glenn A.; Rooney, William D.; Wang, D.J.; DeVos, Soren; Pollaro, Jim; Triplett, William; Lott, Donovan J.; Senesac, Claudia; Daniels, Michael J.; Willcocks, Rebecca J.; Byrne, Barry; Russman, Barry S.; Finkel, Richard S.; Sweeney, H. Lee; Vandenborne, Krista

doi:10.1016/j.nmd.2011.06.801

Cited by 1 publication

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“…The data used in this investigation were generated as part of the multisite ImagingDMD study (NCT01484678) which was approved by the Institutional Review Boards at three sites (University of Florida, Oregon Health & Science University, Children's Hospital of Philadelphia). The MR and functional protocols for ImagingDMD have been previously described 8,25,26 . Briefly, 180 individuals with DMD aged 4–18 years were recruited between 2010 and 2018, and each individual participated in annual follow‐up visits.…”

Section: Subjects/materials and Methodsmentioning

confidence: 99%

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Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy

Willcocks,

Barnard,

Daniels

et al. 2023

Ann Clin Transl Neurol

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ObjectiveMagnetic resonance (MR) measures of muscle quality are highly sensitive to disease progression and predictive of meaningful functional milestones in Duchenne muscular dystrophy (DMD). This investigation aimed to establish the reproducibility, responsiveness to disease progression, and minimum clinically important difference (MCID) for multiple MR biomarkers at different disease stages in DMD using a large natural history dataset.MethodsLongitudinal MR imaging and spectroscopy outcomes and ambulatory function were measured in 180 individuals with DMD at three sites, including repeated measurements on two separate days (within 1 week) in 111 participants. These data were used to calculate day‐to‐day reproducibility, responsiveness (standardized response mean, SRM), minimum detectable change, and MCID. A survey of experts was also performed.ResultsMR spectroscopy fat fraction (FF), as well as MR imaging transverse relaxation time (MRI‐T2), measures performed in multiple leg muscles, and had high reproducibility (Pearson's R > 0.95). Responsiveness to disease progression varied by disease stage across muscles. The average FF from upper and lower leg muscles was highly responsive (SRM > 0.9) in both ambulatory and nonambulatory individuals. MCID estimated from the distribution of scores, by anchoring to function, and via expert opinion was between 0.01 and 0.05 for FF and between 0.8 and 3.7 ms for MRI‐T2.InterpretationMR measures of FF and MRI T2 are reliable and highly responsive to disease progression. The MCID for MR measures is less than or equal to the typical annualized change. These results confirm the suitability of these measures for use in DMD and potentially other muscular dystrophies.

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Section: Subjects/materials and Methodsmentioning

confidence: 99%

“…The MR and functional protocols for ImagingDMD have been previously described. 8 , 25 , 26 Briefly, 180 individuals with DMD aged 4–18 years were recruited between 2010 and 2018, and each individual participated in annual follow‐up visits. The duration of follow‐up was 0–8 years, with a median of 4 years.…”

Section: Subjects/materials and Methodsmentioning

confidence: 99%