Abstract Details

Title Clinically meaningful change on the 100 meter timed test in neuromuscular diseases

Topic Neuro-rehabilitation

Presentation(s) S33 - Neuro-rehabilitation: Brain and Neuromuscular Recovery (1:22 PM-1:33 PM)

Poster/Presentation
Number 003

Background The 100 meter timed test (100m) is a fixed distance ambulatory assessment used to quantify maximal ambulatory ability in children and adults. Subjects are encouraged to run, if safe, to achieve their fastest time. Change in ambulatory ability is a clinically meaningful outcome used across clinical trials for children and adults. A shorter, fixed distance test allowing maximal performance has the potential to reduce variability in performance over time.

Objective The purpose of our study was to establish the minimal detectable change (MDC) of the 100m in muscular dystrophies to better interpret results over time and evaluate its utility to predict loss of ambulation.

Design/Methods Subjects with Duchenne muscular dystrophy (DMD) or a limb girdle muscular dystrophy (LGMD) completed the 100m during at least 1 study visit. The time to complete the test was recorded and a percent predicted was calculated based on age, gender, and body mass index.

Results The DMD cohort included 160 boys (mean age: 7.3 +/- 2.5 years, range 3.4 to 14.7 years) and LGMD cohort included 85 subjects (mean age: 19.6 +/- 11.6 years, range 2.9 to 54.4 years). A subset (N=71 DMD; N= 28 LGMD) completed the 100m up to 3 years post baseline. The MDC for the 100m was calculated as 1/3 the standard deviation of baseline values as well as the standard error of measurement (SEM) approach. Using the MDC 60% of our longitudinal cohort demonstrated a significant decline in ambulation over 12 months. A 100m time of less than 115 seconds, or 25% of predicted, emerged as a critical value for predicting loss of ambulation in DMD. Differences in the effect of LGMD subtype on trajectory of 100m will be discussed.

Conclusions The 100 meter timed test can be implemented in both pediatric and adult populations and can measure meaningful changes over time.

Authors/Disclosures
Lindsay N. Alfano, PT (Nationwide Children'S Hospital) PRESENTER	Ms. Alfano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ATOM International, Ltd (Amicus Therapeutics, Catabasis, Genethon, Italfarmaco, NS Pharma, Pfizer, PTC Therapeutics). Ms. Alfano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Novartis Gene Therapies. The institution of Ms. Alfano has received research support from Sarepta Therapeutics. The institution of Ms. Alfano has received research support from Audentes Therapeutics/Astellas Gene Therapies. Ms. Alfano has received intellectual property interests from a discovery or technology relating to health care.
Natalie F. Miller, PT	Dr. Miller has received personal compensation for serving as an employee of ATOM International. Dr. Miller has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Casimir.
	No disclosure on file
	No disclosure on file
	No disclosure on file
Chang-Yong Tsao, MD, FAAN (Nationwide children's hospital)	No disclosure on file
Louise R. Rodino-Klapac (Sarepta Therapeutics)	Ms. Rodino-Klapac has received personal compensation for serving as an employee of Sarepta Therapeutics, Inc.. Ms. Rodino-Klapac has received stock or an ownership interest from Sarepta Therapeutics. Ms. Rodino-Klapac has received intellectual property interests from a discovery or technology relating to health care.
Megan A. Waldrop, MD (Nationwide Children's Hospital)	Dr. Waldrop has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sarepta Therapeutics. Dr. Waldrop has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapies.
Kevin M. Flanigan, MD, FAAN (Nationwide CHildrens Hospital)	Dr. Flanigan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Apic Bio. Dr. Flanigan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AavantiBio. Dr. Flanigan has stock in 4D Molecular Therapeutics. The institution of Dr. Flanigan has received research support from Abeona Therapeutics. The institution of Dr. Flanigan has received research support from Sarepta Therapeutics. The institution of Dr. Flanigan has received research support from Astellas Therapeutics. Dr. Flanigan has received intellectual property interests from a discovery or technology relating to health care.
Jerry R. Mendell, MD, FAAN (The Research Institute at Nationwide Children's Hospital)	Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.
Linda P. Lowes, PT PhD	The institution of Ms. Lowes has received research support from Sarepta Therapeutics.

��ɫ��

��ɫ��