dc.contributor.author
Dorsch, Eva-Maria
dc.contributor.author
Röhling, Hanna Marie
dc.contributor.author
Zocholl, Dario
dc.contributor.author
Hafermann, Lorena
dc.contributor.author
Paul, Friedemann
dc.contributor.author
Schmitz-Hübsch, Tanja
dc.date.accessioned
2024-05-31T12:15:14Z
dc.date.available
2024-05-31T12:15:14Z
dc.identifier.uri
https://refubium.fu-berlin.de/handle/fub188/43717
dc.identifier.uri
http://dx.doi.org/10.17169/refubium-43432
dc.description.abstract
Background: This study relates to emerging concepts of appropriate trial designs to evaluate effects of intervention on the accumulation of irreversible disability in multiple sclerosis (MS). Major starting points of our study are the known limitations of current definitions of disability progression by rater-based clinical assessment and the high relevance of gait and balance dysfunctions in MS. The study aims to explore a novel definition of disease progression using repeated instrumental assessment of relevant motor functions performed by patients in their home setting.
Methods: The study is a prospective single-center observational cohort study with the primary outcome acquired by participants themselves, a home-based assessment of motor functions based on an RGB-Depth (RGB-D) camera, a camera that provides both depth (D) and color (RGB) data. Participants are instructed to perform and record a set of simple motor tasks twice a day over a one-week period every 6 months. Assessments are complemented by a set of questionnaires. Annual research grade assessments are acquired at dedicated study visits and include clinical ratings as well as structural imaging (MRI and optical coherence tomography). In addition, clinical data from routine visits is provided semiannually by treating neurologists. The observation period is 24 months for the primary endpoint with an additional clinical assessment at 27 month to confirm progression defined by the Expanded Disability Status Scale (EDSS). Secondary analyses aim to explore the time course of changes in motor parameters and performance of the novel definition against different alternative definitions of progression in MS. The study was registered at Deutsches Register für Klinische Studien (DRKS00027042).
Discussion: The study design presented here investigates disease progression defined by marker-less home-based assessment of motor functions against 3-month confirmed disease progression (3 m-CDP) defined by the EDSS. The technical approach was chosen due to previous experience in lab-based settings. The observation time per participant of 24, respectively, 27 months is commonly conceived as the lower limit needed to study disability progression. Defining a valid digital motor outcome for disease progression in MS may help to reduce observation times in clinical trials and add confidence to the detection of progression events in MS.
en
dc.rights.uri
https://creativecommons.org/licenses/by/4.0/
dc.subject
multiple sclerosis
en
dc.subject
disease progression
en
dc.subject
outcome measures
en
dc.subject
motor performance
en
dc.subject
digital biomarker
en
dc.subject.ddc
600 Technik, Medizin, angewandte Wissenschaften::610 Medizin und Gesundheit::610 Medizin und Gesundheit
dc.title
Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study
dc.type
Wissenschaftlicher Artikel
dcterms.bibliographicCitation.articlenumber
1258635
dcterms.bibliographicCitation.doi
10.3389/fneur.2023.1258635
dcterms.bibliographicCitation.journaltitle
Frontiers in Neurology
dcterms.bibliographicCitation.originalpublishername
Frontiers Media SA
dcterms.bibliographicCitation.volume
14
refubium.affiliation
Charité - Universitätsmedizin Berlin
refubium.resourceType.isindependentpub
no
dcterms.accessRights.openaire
open access
dcterms.bibliographicCitation.pmid
37881311
dcterms.isPartOf.eissn
1664-2295