Abstract Details

Title Identifying Digital Biomarkers for the Self-Monitoring of Patients Living with Generalized Myasthenia gravis: a Proof of Concept

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 10-007

Objective To identify clinically meaningful digital biomarkers (dBMKs) intended to track symptoms and signs among people living with generalized myasthenia gravis (gMG) and to prepare the development of a software medical device for the patients’ unsupervised digital self-assessment.

Background Generalized myasthenia gravis (gMG) is characterized by fluctuating manifestations and heterogeneity in fatigue and muscle group weakness. Literature pinpoints a lack of objective measurement tools for the self-assessment of gMG symptoms outside office visits, resulting in different perceptions between physicians and patients on the impact of the disease.

Design/Methods We used a Double Diamond methodology to identify dBMKs of interest for gMG patients and evaluate their relevance for clinicians through qualitative interviews. Unmet needs and dBMKs concepts were prioritized during a technical Proof-of-Concept (PoC) study. The acceptability of the digital device was assessed with prototype-supported interviews.

Results

Interviews with 27 patients (France, USA) confirmed the feasibility of dBMKs to measure gMG-related neurological functions. All were interested in tracking symptoms. 9 international MG experts were interested in assessing gMG symptomology between office visits with clinically meaningful dBMKs. A PoC study on healthy subjects, performed under different conditions, confirmed the feasibility of analyzing raw data collected by the smartphone’s sensors. 5 digital active measures inspired by clinical gold standards and relying on the smartphone’s sensors were selected with their related dBMKs (ptosis, breathing, dysarthria, upper and lower extremities functions). Patient-reported outcome measures (daily activities, pain, sleep, depression, quality of life) will be incorporated as e-questionnaires.

Conclusions We perceived a need for an unsupervised patient self-assessment digital solution to monitor symptomatology outside office visits. In 2023, development of ME&MG, a smartphone solution, will begin. It aims to inform and improve communication between patients and their care teams. International multicenter studies comparing ME&MG to clinical gold standards including the Quantitative Myasthenia Gravis score, will be deployed.

Authors/Disclosures
Clarissa Gorin, Other (Ad Scientiam) PRESENTER	Dr. Gorin has received personal compensation for serving as an employee of Ad Scientiam.
Pascal Laforet (Hopital Pitie Salpetriere)	No disclosure on file
David Orlikowski, MD (Hospital R Poincare)	Dr. Orlikowski has nothing to disclose.
Guillaume Nicolas (AP-HP)	Guillaume Nicolas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson & Johnson. Guillaume Nicolas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Guillaume Nicolas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Guillaume Nicolas has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson.
Helene Prigent (Hopital Raymond Poincaré - APHP)	Helene Prigent has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus therapeutics SAS. Helene Prigent has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Lowenstein Medical. Helene Prigent has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Pfizer. Helene Prigent has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SOS Oxygen. Helene Prigent has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for ASV Santé- Oxylis.
Edouard Berling	Edouard Berling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Edouard Berling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Edouard Berling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Edouard Berling has received personal compensation in the range of $500-$4,999 for serving as a Consultant for LFB.
Ghislain N'Dah-Sekou	No disclosure on file
	No disclosure on file
Juliette Narcy	No disclosure on file
	No disclosure on file
Emma Touré Cuq (Ad Scientiam)	No disclosure on file
Martin Keller (Alexion GmbH)	No disclosure on file
Brian Werneburg (Stoke Therapeutics)	Mr. Werneburg has received personal compensation for serving as an employee of Stoke Therapeutics.
Emrah Aras (Alexion Pharmaceuticals Inc.)	No disclosure on file
Michael G. Benatar, MBChB, DPhil, FAAN (University of Miami)	Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. The institution of Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Arrowhead. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Benatar has received personal compensation in the range of $0-$499 for serving as a Consultant for Annexon. Dr. Benatar has received personal compensation in the range of $0-$499 for serving as a Consultant for UniQure. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cartesian. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Horizon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eli Lilly. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for CorEvitas. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Canopy. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alaunos. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Prilenia. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alector. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for BMS. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Woolsey. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Benatar has received intellectual property interests from a discovery or technology relating to health care.

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