Remote assessment of myotonic dystrophy type 1: A feasibility study

Hamel, Johanna; Creigh, Peter D.; Dekdebrun, Jeanne; Eichinger, Katy; Thornton, Charles A.

doi:10.1002/mus.27559

Cited by 3 publications

(3 citation statements)

References 16 publications

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“…Positive correlation between strength of the knee extensor muscles and both count of steps climbed and sit to stand [ 147 ] Ipod HD Amplitude of thoracic and pelvic trunk movements was significantly greater in participants with HD. Individuals with HD demonstrated rapid movements with varying amplitudes that continuously increased without stabilizing [ 66 ] Jamar Plus Digital; JLW Instruments DM1 Grip strength demonstrated strong correlations with self-reported inventories of upper [ 38 ] JiBuEn gait analysis system SCA There were significant differences in stride length, velocity, supporting-phase percentage, and swinging- phase percentage between the SCA group and the gait control group. Negative correlation between Velocity and ICARS and SARA scores.…”

Section: Resultsmentioning

confidence: 99%

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The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

Poleur

Markati

Servais

2023

Orphanet J Rare Dis

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Developing drugs for rare diseases is challenging, and the precision and objectivity of outcome measures is critical to this process. In recent years, a number of technologies have increasingly been used for remote monitoring of patient health. We report a systematic literature review that aims to summarize the current state of progress with regard to the use of digital outcome measures for real-life motor function assessment of patients with rare neurological diseases. Our search of published literature identified 3826 records, of which 139 were included across 27 different diseases. This review shows that use of digital outcome measures for motor function outside a clinical setting is feasible and employed in a broad range of diseases, although we found few outcome measures that have been robustly validated and adopted as endpoints in clinical trials. Future research should focus on validation of devices, variables, and algorithms to allow for regulatory qualification and widespread adoption.

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Section: Resultsmentioning

confidence: 99%

“…Three used inertial technology. The fourth showed the feasibility of remote video assessment of timed-up-and-go and a digitally measured hand grip strength [ 38 ].…”

Section: Resultsmentioning

confidence: 99%

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

Poleur

Markati

Servais

2023

Orphanet J Rare Dis

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“…The report in this issue by Hamel and colleagues 15 adds to this growing body of evidence for remote monitoring in neuromuscular care and research. Patients with myotonic dystrophy type 1 (DM1) face many of the same barriers to participating in research as those with other neuromuscular disorders; the time, travel, and lost income required to attend centralized study visits can exclude otherwise eligible individuals.…”

mentioning

confidence: 96%

Remote patient monitoring in neuromuscular disease

Geronimo

2022

Muscle and Nerve

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When faced with telemedicine caseloads much lower than those in the early coronavirus disease 2019 (COVID-19) pandemic, practitioners of neuromuscular medicine might be tempted to think of digital health as a passing trend of the early 2020s. However, the use of digital, Internetconnected devices for monitoring patient outcomes has seen sustained growth over the past few years and continues to flourish. Digital health, or "computing platforms, connectivity, software, and sensors for health care and related uses," 1 encompasses a broadly expressed set of tools through which patients receive care. Its potential in neurology has been proposed to be two-fold: (1) to supplement existing practices in ways that are objective, valid, and less burdensome for patients; and (2) as a complete re-imagination of the neurologic exam, its players, and the environment in which it is conducted. 1 Synchronous telemedicine consultation represents just one such implementation of digital health. Digital biomarkers, developed at the intersection of medicine, materials science, and engineering, are another rapidly expanding field in this landscape. Digital biomarkers use a sensor to collect objective data on a biological, anatomical, or physiological parameter, often paired with algorithms that process these data into meaningful outcome measures. 2 These devices can

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Rehabilitation technology in assessment and treatment of arm and hand function in myotonic dystrophy type 1: A single subject experimental design study

Fossmo,

Ørstavik,

Gurandsrud

et al. 2024

Journal of Neuromuscular Diseases

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Background: Myotonic Dystrophy type 1 (DM1) is a slowly progressive hereditary neuromuscular disorder affecting several organ systems including the musculoskeletal system. Objective: To examine the effects of rehabilitation technology on arm and hand function in the assessment and treatment of adults with DM1 with moderate to severe muscle impairment. Methods: A single subject experimental design study (SSED) with 6 participants. The assessments were done weekly through remote study assessments using the video-conference system Whereby®. Participants performed the nine-hole peg test (NHPT), active range of motion (AROM) of the shoulder and patient reported outcome measures (PROMS). Three participants also measured grip and pinch strength and performed the Nut and Bolt task. The intervention was a three-week in-patient rehabilitation stay with the use of rehabilitation technology for exercising arm and hand function. Exercises were performed in AMADEO and the ArmeoSenso. The participants exercised the upper extremities for 30 min 5 times a week. Results: Improvement on the NHPT test on the dominant hand were found for five of the six participants. Three of six improved on the non-dominant hand and in AROM. Two of the three participants that measured pinch and grip strength improved, and all three improved on the Nut and Bolt task. Self- reported function, myotonia and fatigue remained stable. All participants were able to participate in video assessments. Conclusions: Impairment in arm- and hand function affects independence in people with DM1.Our findings suggest that exercising arm- and hand using rehabilitation technology may have a positive effect on function measured by dexterity, strength, and movement in DM1. There is a need to study the effect of exercise on arm- and hand function in DM1 in larger studies. The use of video-consultations can be a supplement in assessment of people with neuromuscular conditions in research and clinical practise.

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Remote assessment of myotonic dystrophy type 1: A feasibility study

Cited by 3 publications

References 16 publications

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review

Remote patient monitoring in neuromuscular disease

Rehabilitation technology in assessment and treatment of arm and hand function in myotonic dystrophy type 1: A single subject experimental design study

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