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Abstract Details

Psychometric Validation of a Modified Unified Multiple System Atrophy Rating Scale
Neuromuscular and Clinical Neurophysiology (EMG)
S43 - Autonomic Disorders (4:42 PM-4:54 PM)
002

The UMSARS was designed to evaluate the signs and symptoms of multiple system atrophy (MSA) that progress over a patient’s course of disease. A subset of 9-items from the UMSARS was proposed for use as a clinically meaningful and sensitive measure of disease progression in a clinical trial conducted over 1-year. Psychometric validation was conducted to inform measurement properties of this 9-item selection (speech, cutting food and handling utensils, dressing, hygiene, walking, urinary function, arising from chair, posture, gait).

To evaluate the measurement properties of a 9-item modified Unified Multiple System Atrophy Rating Scale (UMSARS).

The data used for this analysis was obtained from the M-STAR phase 3 clinical trial in participants with MSA (NCT03952806). The psychometric properties evaluated were data acceptability (floor and ceiling effects), internal consistency reliability, test-retest reliability, convergent validity, and responsiveness (cross-sectional evaluation).

 

Floor or ceiling effects were not observed. Strong internal consistency reliability was demonstrated (αtotal = 0.79) and item-to-total relationships were adequate (r ≥ 0.50) for all items, except urinary function item [r=0.31]). The intra-class correlation coefficient (ICC) indicated strong test-retest reliability (ICC=0.93). Correlations between the modified UMSARS and other validated MSA scales were as follows: r=0.64 with MSA-QoL motor domain p<0.0001; r=-0.41 with SF-36 physical component summary p<0.0001; r=0.27 with Zarit Burden Interview p<0.0001; r=0.23 with PQoL carers p<0.0001; r=0.07 with COMPASS 5-Domain SELECT, p=0.2217. Mean scores for each item increased with disease severity (as assessed by UMSARS-IV) (ANOVA, p<.05), except for the urinary function item.

This 9-item modified UMSARS demonstrated adequate evidence of psychometric validity.  While removal of the single non-motor item (urinary item) resulted in a measure with higher responsiveness, consistency, and reliability, it should be considered that this modification eliminates a core non-motor dysfunction within this heterogenous disease.

Authors/Disclosures
Michele Potashman, PhD (Biohaven)
PRESENTER
Dr. Potashman has received personal compensation for serving as an employee of Biohaven Pharmaceuticals.
No disclosure on file
Susan Durham Susan Durham has received personal compensation for serving as an employee of Biohaven. Susan Durham has stock in Biohaven.
Victoria Wirtz No disclosure on file
Gilbert J. L'Italien Gilbert J. L'Italien has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Gilbert J. L'Italien has stock in biohaven pharmaceuticals.
Vladimir Coric Vladimir Coric has received personal compensation for serving as an employee of Biohaven. Vladimir Coric has received personal compensation in the range of $1,000,000+ for serving as an officer or member of the Board of Directors for Bioahven. Vladimir Coric has stock in Biohaven. Vladimir Coric has received intellectual property interests from a discovery or technology relating to health care.
No disclosure on file
Horacio C. Kaufmann, MD, FAAN (NYU Langone Health - NYU Dysautonomia Center) Dr. Kaufmann has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Theravance. Dr. Kaufmann has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lilly USA. Dr. Kaufmann has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda Pharmaceuticals. Dr. Kaufmann has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ono Pharma UK Ltd. Dr. Kaufmann has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vaxxinity. Dr. Kaufmann has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Spinger. The institution of Dr. Kaufmann has received research support from Biogen. Dr. Kaufmann has received publishing royalties from a publication relating to health care.
Phillip A. Low, MD, FAAN (Mayo Clinic) Dr. Low has nothing to disclose.
Nikolaus McFarland, MD, PhD, FAAN (University of Florida) Dr. McFarland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sutter Health. The institution of Dr. McFarland has received research support from NIH. Dr. McFarland has received publishing royalties from a publication relating to health care.
No disclosure on file
Patricio Millar Vernetti, MD (NYU Dysautonomia Center) The institution of Dr. Millar Vernetti has received research support from NIH. The institution of Dr. Millar Vernetti has received research support from Theravance. Dr. Millar Vernetti has received personal compensation in the range of $500-$4,999 for serving as a Speaker with WPC.
Huw R. Morris, MD, PhD Dr. Morris has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Morris has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Morris has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Personal injury / Negligence cases. The institution of Dr. Morris has received research support from Parkinson's UK. The institution of Dr. Morris has received research support from Michael J Fox Foundartion. The institution of Dr. Morris has received research support from Cure Parkinson's Trust. The institution of Dr. Morris has received research support from PSP Association. The institution of Dr. Morris has received research support from CBD Solutions. The institution of Dr. Morris has received research support from Drake Foundation. The institution of Dr. Morris has received research support from Medical Research Council. Dr. Morris has received intellectual property interests from a discovery or technology relating to health care. Dr. Morris has a non-compensated relationship as a Scientific Advisory Board with Cure PSP that is relevant to AAN interests or activities. Dr. Morris has a non-compensated relationship as a Scientific Advisory Board with Parkinson's UK that is relevant to AAN interests or activities.
Werner Poewe, MD (University Hospital of Neurology) Werner Poewe, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Affiris . Werner Poewe, MD has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alterity, AbbVie, BIAL, Biogen, Britannia, Lilly, Lundbeck, Neuroderm, Neurocrine, Denali Pharmaceuticals, ,Roche, Takeda, UCB and Zambon . Werner Poewe, MD has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for AbbVie, Britannia, BIAL, STADA, Lundbeck, Zambon. The institution of Werner Poewe, MD has received research support from MJFF; EU Horizon programme. Werner Poewe, MD has a non-compensated relationship as a Committee member with Movement Disorder Society that is relevant to AAN interests or activities.
Klaus Seppi, MD Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ONO . Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BIAL. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AbbVie. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Bial. Dr. Seppi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Teva. The institution of Dr. Seppi has received research support from MJFF. The institution of Dr. Seppi has received research support from EU. The institution of Dr. Seppi has received research support from FWF Austrian Science Fund,.
Wolfgang Singer, MD (Mayo Clinic) Dr. Singer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amneal. Dr. Singer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UniQure. Dr. Singer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven. Dr. Singer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Theravance. The institution of Dr. Singer has received research support from NIH. The institution of Dr. Singer has received research support from FDA. The institution of Dr. Singer has received research support from Michael J. Fox Foundation. Dr. Singer has received intellectual property interests from a discovery or technology relating to health care.
Steven Vernino, MD, PhD, FAAN (UT Southwestern Medical Center) Dr. Vernino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for antag. Dr. Vernino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. Dr. Vernino has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Vernino has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyverna. Dr. Vernino has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Autonomic Neuroscience (Elsevier). The institution of Dr. Vernino has received research support from Takeda. The institution of Dr. Vernino has received research support from NIH/NHLBI. The institution of Dr. Vernino has received research support from Dysautonomia International. Dr. Vernino has received personal compensation in the range of $10,000-$49,999 for serving as a Content Expert Consultant with Office of Inspector General for Medicare. Dr. Vernino has a non-compensated relationship as a Board of Directors with American Autonomic Society that is relevant to AAN interests or activities.
Irfan Qureshi, MD (Biohaven Pharmaceuticals) Dr. Qureshi has received personal compensation for serving as an employee of Biohaven. Dr. Qureshi has stock in Biohaven Pharmaceuticals.