Abstract Details

Title ME&MGTM Digital Biomarkers Correlate With Equivalent MG-ADL and MG-QoL15r Sub-items Scores

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P9 - Poster Session 9 (5:00 PM-6:00 PM)

Poster/Presentation
Number 9-015

Objective To describe the correlation between ME&MGTM digital biomarkers (dBMKs) and corresponding MG-ADL and MG-QoL15r sub-items from the decentralized ME&MGopenTM study (NCT05566964).

Background Symptoms of generalized Myasthenia gravis (gMG), a rare auto-immune neuromuscular disorder characterized by fatigable muscle weakness, are routinely captured via patient-reported outcomes (PROs) questionnaires. MG-ADL and MG-QoL15r assess daily activities with 8 questions scored from 0-3, and quality of life with 15 questions scored from 0-2; higher scores indicate higher severity and worse quality of life, respectively. DBMKs provide objective measures for patients to assess their symptoms, at home, by performing smartphone-based active tests. ME&MGTM evaluates five gMG symptoms: ptosis, dysarthria, respiratory function, upper and lower limb weakness mirrored in 5 MG-ADL and 3 MG-QOL15r items. In ME&MGopen, gMG patients performed ME&MGTM tests for one year.

Design/Methods ME&MGTM dBMKS MyEyelids, MyVoice, MyBreathing, MyArms and MyLegs respectively measure Margin-to-reflex-distance-1, dysarthria, sustained phonation, holding duration and number of sit-to-stand, corresponding to sub-items of MG-ADL/MG-QoL15r. Completed tests were reviewed for quality (inaccurate dBMKs values and agreement with human measurements) before analysis. The relationship between dBMKs and sub-items was determined using mixed models analyses.

Results 156 (67.9% female, mean age 58.7±15.8, mean disease duration 11.2±12.3) participants completed digital tests and questionnaires, monthly. A total of 9,106 evaluations were collected and 90.4% of them were of good quality. DBMKs scores for ptosis, breathing, and limb weakness decreased with increasing symptom severity assessed by PROs. Dysarthria scores increased with symptom severity. Ptosis, dysarthria and limb weakness scores showed significant associations (all p<0.019) with aforementioned sub-items of MG-ADL/MG-QoL-15r scores.

Conclusions Significant associations between ME&MGTM dBMKs and clinical assessments support their potential use in clinical settings to track gMG manifestations. These findings highlight the importance of the ongoing validation study DOMYA (NCT05564936).

Authors/Disclosures
Clarissa Gorin, Other (Ad Scientiam) PRESENTER	Dr. Gorin has received personal compensation for serving as an employee of Ad Scientiam.
Carolina Barnett Tapia, MD	Dr. Barnett Tapia has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Barnett Tapia has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD pharma. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Barnett Tapia 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. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Barnett Tapia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. The institution of Dr. Barnett Tapia has received research support from Department of defense. The institution of Dr. Barnett Tapia has received research support from Muscular dystrophy canada. The institution of Dr. Barnett Tapia has received research support from MgNet. The institution of Dr. Barnett Tapia has received research support from NIH. The institution of Dr. Barnett Tapia has received research support from Cartesian. The institution of Dr. Barnett Tapia has received research support from Alexion. Dr. Barnett Tapia has received intellectual property interests from a discovery or technology relating to health care.
Sophie Lehnerer	Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Sophie Lehnerer has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Hormosan.
Séverine Bieuvelet	Séverine Bieuvelet has received personal compensation for serving as an employee of Ad Scientiam.
Loic Carment (Ad Scientiam)	Loic Carment has received personal compensation for serving as an employee of Ad Scientiam.
Natacha Pesic Heuvrard, MD	Miss Pesic Heuvrard has received personal compensation for serving as an employee of Ad Scientiam.
Dellini Ravindra (Ad Scientiam)	Dellini Ravindra has received personal compensation for serving as an employee of Ad Scientiam.
Noura Sellami	Miss Sellami has nothing to disclose.
Bhaskar Dutta, PhD (Alexion)	Dr. Dutta has received personal compensation for serving as an employee of AstraZeneca. An immediate family member of Dr. Dutta has received personal compensation for serving as an employee of Sanofi.
Benjamin Yungher, PhD (Alexion)	Dr. Yungher has received personal compensation for serving as an employee of Alexion AstraZeneca Rare Disease.
Saad Zinai	Saad Zinai has received personal compensation for serving as an employee of AD Scientiam. Saad Zinai has stock in Ad Scientiam.
James F. Howard, Jr., MD, FAAN (The University of North Carolina, Dept of Neurology, CB 7025)	Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMGEN (Horizon Therapeutics). Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven Ltd. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CorEvitas. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck EMD Serono. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NMD Pharma. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for H. Lundbeck A/S. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Seismic Therapeutics. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vor Biopharma. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for MJH LifeSci. Dr. Howard has or had stock in Johnson & Johnson dividends.Dr. Howard has or had stock in Pfizer dividends. An immediate family member of Dr. Howard has or had stock in GlaxoSmithKline dividends.Dr. Howard has or had stock in Bristol Myer Squbb dividends.Dr. Howard has or had stock in AbbieVie Inc. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Cartestian Therapeutics. The institution of Dr. Howard has received research support from NMD Pharma. The institution of Dr. Howard has received research support from Ad Scientiam. The institution of Dr. Howard has received research support from Merck EMD Serono. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.

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