ME&MGTM Digital Biomarkers Correlate With Equivalent MG-ADL and MG-QoL15r Sub-items Scores
Carolina Barnett-Tapia1, Sophie Lehnerer2, Séverine Bieuvelet3, Loïc Carment3, Clarissa Gorin3, Natacha Pesic-Heuvrard3, Dellini Ravindra3, Noura Sellami3, Bhaskar Dutta4, Benjamin Yungher4, Saad Zinaï3, James Howard5
1Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, Ontario, Canada, 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, AstraZeneca Rare Disease, Boston, MA, USA, 5University of North Carolina, Department of Neurology, Chapel Hill, NC, USA
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).
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