Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich’s ataxia
| Autor: | David A. Lynch, Michelle L. Krishnan, Mary Bylo, Arne Mueller, Holger Hoefling, Jens Praestgaard, Elaine Paterson, Christian Rummey, McKenzie Wells, C J Malanga, Avery McIntosh, Meredith Schultz |
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| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Male
medicine.medical_specialty congenital hereditary and neonatal diseases and abnormalities Neurology Ataxia Activities of daily living Adolescent Population Diagnostic Techniques Neurological Neurosciences. Biological psychiatry. Neuropsychiatry Severity of Illness Index Physical medicine and rehabilitation Rating scale Activities of Daily Living Medicine Humans education Child RC346-429 Research Articles Balance (ability) education.field_of_study business.industry General Neuroscience Gait Test (assessment) Cross-Sectional Studies Friedreich Ataxia Disease Progression Feasibility Studies Female Neurology (clinical) Neurology. Diseases of the nervous system medicine.symptom business Research Article RC321-571 |
| Zdroj: | Annals of Clinical and Translational Neurology, Vol 8, Iss 9, Pp 1845-1856 (2021) Annals of Clinical and Translational Neurology |
| ISSN: | 2328-9503 |
| Popis: | Background Friedreich's ataxia is an inherited, progressive, neurodegenerative disease that typically begins in childhood. Disease severity is commonly assessed with rating scales, such as the modified Friedreich's Ataxia Rating Scale, which are usually administered in the clinic by a neurology specialist. Objective This study evaluated the utility of home-based, self-administered digital endpoints in children with Friedreich's ataxia and unaffected controls and their relationship to standard clinical rating scales. Methods In a cross-sectional study with 25 participants (13 with Friedreich's ataxia and 12 unaffected controls, aged 6-15 years), home-based digital endpoints that reflect activities of daily living were recorded over 1 week. Domains analyzed were hand motor function with a digitized drawing, automated analysis of speech with a recorded oral diadochokinesis test, and gait and balance with wearable sensors. Results Hand-drawing and speech tests were easy to conduct and generated high-quality data. The sensor-based gait and balance tests suffered from technical limitations in this study setup. Several parameters discriminated between groups or correlated strongly with modified Friedreich's Ataxia Rating Scale total score and activities of daily living total score in the Friedreich's ataxia group. Hand-drawing parameters also strongly correlated with standard 9-hole peg test scores. Interpretation Deploying digital endpoints in home settings is feasible in this population, results in meaningful and robust data collection, and may allow for frequent sampling over longer periods of time to track disease progression. Care must be taken when training participants, and investigators should consider the complexity of the tasks and equipment used. |
| Databáze: | OpenAIRE |
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