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Abstract Details

Current Abilities Score (CAS) is a Valid and a Reliable PROM in FSHD: Data From MOVE Natural History Study
Neuromuscular and Clinical Neurophysiology (EMG)
S7 - Updates on Muscle Disorders (4:54 PM-5:06 PM)
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To assess the utility of CAS as a novel patient reported outcome measure

Patient reported outcome measures (PROM) are important to measure both the impact of FSHD on the function and quality of life. If the PROM is also sensitive to change over time then its crucial for planning day-day care as well as recording patient related experiences in both natural history studies and clinical trials.

 

 

 

 
CAS is a patient reported outcome measure used in two large multi-national natural history studies in FSHD, RESOLVE and MOVE. It has six items including functions of face, arms, hands, mobility, chair and bed transfers with a maximum score of 25 points indicating severe disability. Here we report the baseline and longitudinal results for CAS from MOVE study. Various items of validation  and several psychometric analysis were carried out. Baseline characteristics and longitudinal changes were analysed.
Baseline CAS from 437 FSHD patients was available. 209 were female with an average age of 48 yrs (range 5-83yr) and average FCS scale 7.32 (SD±4.05). CAS positively correlated well with FCS (p=0.79, p<0.001), MMT sum score (p=0.74, p<0.001), negatively with Upper extremity Functional index (UEFI) (p= -0.78, p<0.001). CAS also had excellent reliability in both in-person and remote completions (ICC day 1 vs day 14 ICC = 0.979 (n = 15). At 24 months, the percentage change from baseline was 9.8%. Further change over time trajectories and subgroup analysis at 6-12 monthly intervals will be presented.
We present CAS data from a large multi-center FSHD natural history study called MOVE. We propose that CAS is a valid, and a reliable patient reported outcome measure in persons with FSHD. To be able to demonstrate change over time is paramount to clinical trials and in clinic monitoring and to plan interventions.
Authors/Disclosures
Channa A. Hewamadduma, MBBS, PhD, FRCP, MSc (Royal Hallamshire Hospital)
PRESENTER 		Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ARENX. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BIOGEN. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ROCHE. The institution of Dr. Hewamadduma has received research support from Neurocare . The institution of Dr. Hewamadduma has received research support from Sheffield Charitable Trust. Dr. Hewamadduma has received personal compensation in the range of $5,000-$9,999 for serving as a Adult Expert with National Health Service England (NHSE).
Michaela Walker, MPH, CCRP (University of Kansas Medical Center) Miss Walker has nothing to disclose.
Jon Street (Sheffield Teaching Hospitals) The institution of Jon Street has received research support from Sheffield Hospitals Charity.
Natalie Katz, MD, PhD (Duke University) Dr. Katz has nothing to disclose.
Katy Eichinger, PhD, PT, DPT, NCS (University of Rochester) Dr. Eichinger has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fulcrum. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Avidity. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for DyneTherapeutic. Dr. Eichinger has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TRiNDS. The institution of Dr. Eichinger has received research support from Charcot Marie Tooth Association. The institution of Dr. Eichinger has received research support from Muscular Dystrophy Association. Dr. Eichinger has received intellectual property interests from a discovery or technology relating to health care.
Filipe Di Pace, MD Dr. Di Pace has nothing to disclose.
Sandhya Sasidharan, PT Dr. Sasidharan has nothing to disclose.
Linda P. Lowes, PT PhD The institution of Ms. Lowes has received research support from Sarepta Therapeutics.
Cristiane D. Moreno, MD, PhD Dr. Moreno has nothing to disclose.
Jeffrey Statland, MD (University of Kansas Medical Center) Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.