First Cross-sectional Analysis from the ME&MGopen Study: A Decentralized Study on App-based Myasthenia Gravis
Carolina Barnett Tapia1, Sophie Lehnerer2, Laïtissia Ahamada3, Loic Carment3, Kenza Drareni3, Pierre Drouin3, Clarissa Gorin3, Dellini Ravindra3, Emma Touré Cuq3, Alizé Vivès3, Emrah Aras4, Martin Keller5, Saad Zinaï3, James Howard6
1Department of Medicine, Division of Neurology. University of Toronto and University Health Network, 2Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Department of Neurology with Experimental Neurology, Charitéplatz 1, 10117 Berlin, Germany, 3Ad Scientiam, Paris, France, 4Alexion Pharmaceuticals Inc., 5Alexion GmbH, 6The University of North Carolina, Dept of Neurology, CB 7025
Objective:
To describe and assess real-world data (RWD), adherence, user experience, safety, and satisfaction collected using ME&MGopenTM 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&MGopenTM app. ME&MGopenTM, 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&MGopenTM received a high satisfaction rating of 8.5/10.
Conclusions:
Initial findings indicate substantial compliance and overall satisfaction with ME&MGopenTM. Patients’ demographics are consistent with the gMG epidemiology, allowing the remote collection of comprehensive RWD accurately representing gMG patients.