ArticleAnnals of clinical and translational neurology2021
Digital endpoints for self-administered home-based functional assessment in pediatric Friedreich's ataxia.
Article in Annals of clinical and translational neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Progressive scoliosis in a pediatric patient with cerebellar ataxia: surgical challenges and literature review.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Pooled it
- Continuous Movement Monitoring at Home Through Wearable Devices: A Systematic Review.Sensors (Basel, Switzerland) · 2025Pooled it
- The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review.Orphanet journal of rare diseases · 2023Pooled it
- Remote Monitoring in Myasthenia Gravis: Exploring Symptom Variability.Annals of clinical and translational neurology · 2026Trial
- Digital Outcomes of Upper Limb Ataxia Capture Meaningful Longitudinal Change and Treatment Response.Movement disorders : official journal of the Movement Disorder Society · 2025Article
- Speech Differences between Multiple System Atrophy and Parkinson's Disease.Movement disorders clinical practice · 2025Article
- Wearable sensors in paediatric neurology.Developmental medicine and child neurology · 2025Review
- Dietary and lifestyle interventions for the management of hereditary ataxias.Frontiers in nutrition · 2025Review
- Gait characteristics in people with Friedreich ataxia: daily life versus clinic measures.Frontiers in neurology · 2025Article
- At-home wearable-based monitoring predicts clinical measures and biological biomarkers of disease severity in Friedreich's Ataxia.Communications medicine · 2024Article
- Quantitative Gait and Balance Outcomes for Ataxia Trials: Consensus Recommendations by the Ataxia Global Initiative Working Group on Digital-Motor Biomarkers.Cerebellum (London, England) · 2024Review
- Multifeature quantitative motor assessment of upper limb ataxia including drawing and reaching.Annals of clinical and translational neurology · 2024Article
- Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity.Frontiers in pharmacology · 2024Article
- Referent data for investigations of upper limb accelerometry: harmonized data from three cohorts of typically-developing children.Frontiers in pediatrics · 2024Article
- SARANPJ digital medicine · 2023Article
- Natural History and Real-World Data in Rare Diseases: Applications, Limitations, and Future Perspectives.Journal of clinical pharmacology · 2022Review
- Measuring Health-Related Quality of Life With Multimodal Data: Viewpoint.Journal of medical Internet research · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors.
Funding
Abstract
backgroundFriedreich'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.
objectiveThis 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.
methodsIn 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.
resultsHand-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.
interpretationDeploying 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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.