Evidence map›Paper›PMID 38995798›Full record

ArticleJournal of neuromuscular diseases2024

Smartphone-Based Assessment of Mobility and Manual Dexterity in Adult People with Spinal Muscular Atrophy.

Eduardo Arteaga-Bracho, Gautier Cosne, Christoph Kanzler, Angelos Karatsidis, Claudia Mazzà, Joaquin Penalver-Andres, Cong Zhu, Changyu Shen, Kelley Erb M, Maren Freigang and 12 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of neuromuscular diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
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  5. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

22 authors.

Eduardo Arteaga-BrachoBiogen Digital Health, Biogen, Cambridge, MA, USA.
Gautier CosneBiogen Digital Health, Biogen, Cambridge, MA, USA.
Christoph KanzlerBiogen Digital Health, Biogen, Cambridge, MA, USA.
Angelos KaratsidisBiogen Digital Health, Biogen, Cambridge, MA, USA.
Claudia MazzàBiogen Digital Health, Biogen, Cambridge, MA, USA.
Joaquin Penalver-AndresBiogen Digital Health, Biogen, Cambridge, MA, USA.
Cong ZhuBiogen Digital Health, Biogen, Cambridge, MA, USA.
Changyu ShenBiogen Digital Health, Biogen, Cambridge, MA, USA.
Kelley Erb MBiogen Digital Health, Biogen, Cambridge, MA, USA.
Maren FreigangDepartment of Neurology, Dresden University Hospital, Dresden, Germany.
Hanna-Sophie LappDepartment of Neurology, Dresden University Hospital, Dresden, Germany.
Simone ThieleFriedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
Stephan WenningerFriedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
Erik JungDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Susanne PetriDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Markus WeilerDepartment of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Christoph KleinschnitzDepartment of Neurology, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
Maggie C WalterFriedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
René GüntherDepartment of Neurology, Dresden University Hospital, Dresden, Germany.
Nolan CampbellBiogen Digital Health, Biogen, Cambridge, MA, USA.
Shibeshih BelachewBiogen Digital Health, Biogen, Cambridge, MA, USA.
Tim HagenackerDepartment of Neurology, Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: More responsive, reliable, and clinically valid endpoints of disability are essential to reduce size, duration, and burden of clinical trials in adult persons with spinal muscular atrophy (aPwSMA). Objective: The aim is to investigate the feasibility of smartphone-based assessments in aPwSMA and provide evidence on the reliability and construct validity of sensor-derived measures (SDMs) of mobility and manual dexterity collected remotely in aPwSMA. Methods: Data were collected from 59 aPwSMA (23 walkers, 20 sitters and 16 non-sitters) and 30 age-matched healthy controls (HC). SDMs were extracted from five smartphone-based tests capturing mobility and manual dexterity, which were administered in-clinic and remotely in daily life for four weeks. Reliability (Intraclass Correlation Coefficients, ICC) and construct validity (ability to discriminate between HC and aPwSMA and correlations with Revised Upper Limb Module, RULM and Hammersmith Functional Scale - Expanded HFMSE) were quantified for all SDMs. Results: The smartphone-based assessments proved feasible, with 92.1% average adherence in aPwSMA. The SDMs allowed to reliably assess both mobility and dexterity (ICC > 0.75 for 14/22 SDMs). Twenty-one out of 22 SDMs significantly discriminated between HC and aPwSMA. The highest correlations with the RULM were observed for SDMs from the manual dexterity tests in both non-sitters (Typing, ρ= 0.78) and sitters (Pinching, ρ= 0.75). In walkers, the highest correlation was between mobility tests and HFMSE (5 U-Turns, ρ= 0.79). Conclusions: This exploratory study provides preliminary evidence for the usability of smartphone-based assessments of mobility and manual dexterity in aPwSMA when deployed remotely in participants' daily life. Reliability and construct validity of SDMs remotely collected in real-life was demonstrated, which is a pre-requisite for their use in longitudinal trials. Additionally, three novel smartphone-based performance outcome assessments were successfully established for aPwSMA. Upon further validation of responsiveness to interventions, this technology holds potential to increase the efficiency of clinical trials in aPwSMA.

Indexed as

Muscular Atrophy, SpinalSmartphoneAdultDisability EvaluationFeasibility StudiesFemaleHumansMaleMiddle AgedMotor SkillsReproducibility of ResultsaccelerometersdrawingpinchingRemote monitoringturningtypingwalkingwearable sensors

Identifiers

PMID38995798
PMCPMC11380318

What OpenQuestion holds

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Registered trials

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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.