Identifying Digital Biomarkers for the Self-Monitoring of Patients Living with Generalized Myasthenia gravis: a Proof of Concept (P7-8.007)

Abstract

<h3>Objective:</h3> 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. <h3>Background:</h3> 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. <h3>Design/Methods:</h3> 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. <h3>Results:</h3> 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. <h3>Conclusions:</h3> We perceived a need for an unsupervised patient self-assessment digital solution to monitor symptomatology outside office visits. In 2023, development of ME&amp;MG, a smartphone solution, will begin. It aims to inform and improve communication between patients and their care teams. International multicenter studies comparing ME&amp;MG to clinical gold standards including the Quantitative Myasthenia Gravis score, will be deployed. <b>Disclosure:</b> Pascal Laforet has nothing to disclose. David Orlikowski has nothing to disclose. Prof. NICOLAS has nothing to disclose. The institution of Prof. Prigent has received research support from VLM - Patients association for lysosomial diseases. The institution of Prof. Prigent has received research support from ADEP Assitance. Dr. Berling has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Adscientiam. Dr. Berling has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Ghislain N’Dah-Sekou has nothing to disclose. Dr. Gorin has received personal compensation for serving as an employee of Ad Scientiam. Dr. Moreira has received personal compensation for serving as an employee of Ad Scientiam. Mrs. Narcy has received personal compensation for serving as an employee of Ad Scientiam. Dr. Plaud has received personal compensation for serving as an employee of Ad Scientiam. Mrs. Touré Cuq has received personal compensation for serving as an employee of Ad Scientiam . Dr. Keller has received personal compensation for serving as an employee of Alexion. Dr. Keller has stock in Alexion, Roche, Novartis, Celgene, Abbvie, Merck, Ionis, Sanofie, Organon, MSD. Mr. Werneburg has received personal compensation for serving as an employee of Alexion. Dr. Aras has received personal compensation for serving as an employee of Alexion Pharmaceuticals Inc. Dr. Benatar has received personal compensation in the range of $10,000-$49,999 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 $500-$4,999 for serving as a Consultant for Annexon. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UniQure. Dr. Benatar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alector. Dr. Benatar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Benatar 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. Benatar has received intellectual property interests from a discovery or technology relating to health care. Dr. Benatar has received intellectual property interests from a discovery or technology relating to health care.