Remote assessment of myotonic dystrophy type 1: A feasibility study

• Published in: Muscle & nerve Vol. 66; no. 3; pp. 336 - 339
• Main Authors: Hamel, Johanna, Creigh, Peter D., Dekdebrun, Jeanne, Eichinger, Katy, Thornton, Charles A.
• Format: Journal Article
• Language: English
• Published: Hoboken, USA John Wiley & Sons, Inc 01.09.2022; Wiley Subscription Services, Inc
• Subjects: Assessments; Correlation; Cough; Dystrophy; Feasibility Studies; Grip strength; Hand Strength; Humans; Impairment; Muscle function; Muscle, Skeletal; Muscles; Myotonia; Myotonic dystrophy; Myotonic Dystrophy - diagnosis; outcome measures; Patients; Portable equipment; remote assessments; Tablet computers; Telemedicine; Toolkits; myotonic dystrophy; telemedicine; remote assessments; outcome measures; muscle function
• ISSN: 0148-639X; 1097-4598; 1097-4598
• DOI: 10.1002/mus.27559

Introduction/Aims Remote study visits (RSVs) are emerging as important tools for clinical research. We tested the feasibility of using RSVs to evaluate patients with myotonic dystrophy type 1 (DM1), including remote quantitative assessment of muscle function, and we assessed correlations of remote assessments with patient‐reported function. Methods Twenty three subjects with DM1 were consented remotely. Toolkits containing a tablet computer, grip dynamometer, and spirometer were shipped to participants. The tablets were loaded with software for video‐conferencing and questionnaires about functional impairment, patient experience with technology, and willingness to participate in future remote studies. Grip strength, forced vital capacity, peak cough flow, timed‐up‐and‐go (TUG), and grip myotonia (hand opening time) were determined during RSVs. We assessed correlations of remote assessments with patient‐reported outcomes of muscle function and with CTG repeat size. Results All 23 subjects completed RSVs. 95% of participants were able to complete all components of the remote study. All toolkit components were returned upon completion. Grip strength and TUG demonstrated moderate to strong correlations with self‐reported inventories of upper and lower extremity impairment, respectively (ρ = 0.7 and ρ = −0.52). A total of 91% of subjects expressed interest in participating in future RSVs. Discussion Results of this study support the feasibility of using portable devices and video‐conferencing for remote collection of patient‐reported outcomes and quantitative assessment of muscle function in DM1. See Editorial on pages 233‐235 in this issue