Abstract Details

Title First Cross-sectional Analysis from the ME&MGopen Study: A Decentralized Study on App-based Myasthenia Gravis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P4 - Poster Session 4 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-005

Objective To describe and assess real-world data (RWD), adherence, user experience, safety, and satisfaction collected using ME&MGopen^TM app in patients with generalized myasthenia gravis (gMG).

Background gMG is a rare antibody-mediated neuromuscular condition characterized by fluctuations and variations in fatigable muscle weakness. RWD tracking symptoms and impact on patients’ lives over time in these patients are scarce. A decentralized clinical study could address these data gaps and gather valuable insights into patients' experience.

Design/Methods ME&MGopen is a fully decentralized international clinical trial (USA, Canada) that will enroll ≥120 participants diagnosed with acetylcholine receptor antibody positive-gMG. During the 12-month follow-up, socio-demographic, clinical and functional data will be collected remotely from participants, via a monthly completion with the ME&MGopen^TM app. ME&MGopen^TM, a research device, includes digital active tests and e-questionnaires to capture key functional parameters (ptosis, dysarthria, respiratory capacity, upper and lower limb muscle function), depression, pain, quality of life and insomnia.

Results Preliminary analysis from 173 participants (68% females; mean age 58 ± 17 years) showed that 93% of participants under age 40 were female, while women accounted for 53% of participants over 60 years. Participants gMG severity (MGFA disease class) was 18% II; 59% III and 24% IV. Despite 94% reporting a gMG medication (74% in combination), perceived impacts on symptoms varied with first-line treatment (treatment satisfaction score: 7/10; range [1;10]). Most participants (85%) completed the baseline digital assessment and 50% were still active after 5 months. ME&MGopen^TM received a high satisfaction rating of 8.5/10.

Conclusions Initial findings indicate substantial compliance and overall satisfaction with ME&MGopen^TM. Patients’ demographics are consistent with the gMG epidemiology, allowing the remote collection of comprehensive RWD accurately representing gMG patients.

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.
Laïtissia AHAMADA (Ad Scientiam)	No disclosure on file
Loic Carment (Ad Scientiam)	Loic Carment has received personal compensation for serving as an employee of Ad Scientiam.
Kenza Drareni (Ad Scientiam)	Kenza Drareni has nothing to disclose.
Pierre DROUIN (AdScientiam)	Pierre DROUIN has received personal compensation for serving as an employee of AdScientiam.
Dellini Ravindra (Ad Scientiam)	Dellini Ravindra has received personal compensation for serving as an employee of Ad Scientiam.
Emma Touré Cuq (Ad Scientiam)	No disclosure on file
Alizé Vives (Ad Scientiam)	No disclosure on file
Emrah Aras (Alexion Pharmaceuticals Inc.)	No disclosure on file
Martin Keller (Alexion GmbH)	No disclosure on file
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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