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Abstract Details

Standardizing Ultrasound Assessment and Quantification for Use in Duchenne Muscular Dystrophy Clinical Trials
Muscle Disease/Neuromuscular Junction
IN1 - (-)
005
Quantitative ultrasound identifies pathology in dystrophic muscles and thus could serve as a non-invasive tool for assessment of disease severity and progression. However, in order for it to serve a role in clinical trials, the reliability and sensitivity of the technique as performed by individuals without specific ultrasound expertise needs to be determined.
Four non-physicians with no previous ultrasound experience except a brief training session, acquired ultrasound images of six muscle groups in arms and legs of 12 boys with DMD (ages 4-12, mean 7.7 years) and 12 controls (ages 2-12, mean 7.9 years). Quantification of the gray scale levels (GSL) with an image processing program was then performed by a trained radiologist and a research assistant with no expertise, outside of one training session. A physical therapist performed functional assessments.
Intra-rater and inter-rater reliability between sonographers was excellent, with intraclass correlation coefficients (ICCs) of 0.85 and 0.9, respectively. The research assistant GSL interpretation was also closely aligned to that of the radiologist with ICCs of 0.83-0.95 depending on the muscle. Consistent with previously published results, GSL (averaged from all muscles) was higher in DMD than control muscle (p<0.001), distinguished DMD boys from controls with high accuracy (area under the receiver operator curve 0.98) and increased with the North Star Ambulatory Assessment scores in the seven boys with DMD on whom it could be performed (rspearman=-0.78, p=0.04).
Non-physician evaluators with limited training can obtain ultrasound data with high reliability. The GSLs obtained from DMD boys were higher than controls and correlated with functional measures, consistent with previous reports. These findings indicate that quantitative ultrasound has the potential to be employed effectively in multicenter clinical therapeutic trials in Duchenne muscular dystrophy.
Authors/Disclosures

PRESENTER 		No disclosure on file
Craig M. Zaidman, MD (Washington University in St Louis) Dr. Zaidman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for sarepta. The institution of Dr. Zaidman has received research support from Washington University in St Louis. The institution of Dr. Zaidman has received research support from Novartis.
Jim Wu No disclosure on file
Seward B. Rutkove, MD, FAAN Dr. Rutkove has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Myolex, Inc. Dr. Rutkove has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for American Academy of Neuorlogy . The institution of Dr. Rutkove has received research support from NIH. The institution of Dr. Rutkove has received research support from NASA. The institution of Dr. Rutkove has received research support from Blavatnik Family Foundation. Dr. Rutkove has received intellectual property interests from a discovery or technology relating to health care. Dr. Rutkove has received intellectual property interests from a discovery or technology relating to health care. Dr. Rutkove has received publishing royalties from a publication relating to health care. Dr. Rutkove has received publishing royalties from a publication relating to health care. Dr. Rutkove has received personal compensation in the range of $500-$4,999 for serving as a Grant Reviewer with NIH.
Alan Pestronk, MD, FAAN (Washington University in Saint Louis - Neurology) The institution of Dr. Pestronk has received research support from Jain. The institution of Dr. Pestronk has received research support from Fulcrum. The institution of Dr. Pestronk has received research support from Argenyx. The institution of Dr. Pestronk has received research support from NeuroNext.