Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study
BackgroundThis 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...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-10-01
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| Series: | Frontiers in Neurology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fneur.2023.1258635/full |
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| author | Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Dario Zocholl Lorena Hafermann Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch |
| author_facet | Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Dario Zocholl Lorena Hafermann Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch |
| author_sort | Eva-Maria Dorsch |
| collection | DOAJ |
| description | BackgroundThis 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.MethodsThe 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).DiscussionThe 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. |
| first_indexed | 2024-03-11T19:07:28Z |
| format | Article |
| id | doaj.art-a531d71c886247078763cbeb3d6fa0fc |
| institution | Directory Open Access Journal |
| issn | 1664-2295 |
| language | English |
| last_indexed | 2024-03-11T19:07:28Z |
| publishDate | 2023-10-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Neurology |
| spelling | doaj.art-a531d71c886247078763cbeb3d6fa0fc2023-10-10T06:22:33ZengFrontiers Media S.A.Frontiers in Neurology1664-22952023-10-011410.3389/fneur.2023.12586351258635Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective studyEva-Maria Dorsch0Eva-Maria Dorsch1Eva-Maria Dorsch2Eva-Maria Dorsch3Eva-Maria Dorsch4Hanna Marie Röhling5Hanna Marie Röhling6Hanna Marie Röhling7Hanna Marie Röhling8Dario Zocholl9Lorena Hafermann10Friedemann Paul11Friedemann Paul12Friedemann Paul13Friedemann Paul14Friedemann Paul15Tanja Schmitz-Hübsch16Tanja Schmitz-Hübsch17Tanja Schmitz-Hübsch18Tanja Schmitz-Hübsch19Experimental and Clinical Research Center, a Cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité—Universitätsmedizin Berlin, Berlin, GermanyExperimental and Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyMax-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, GermanyDepartment of Neurology, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, GermanyNeuroscience Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, GermanyExperimental and Clinical Research Center, a Cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité—Universitätsmedizin Berlin, Berlin, GermanyExperimental and Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyMax-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, GermanyMotognosis GmbH, Berlin, GermanyInstitute of Biometry and Clinical Epidemiology, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyInstitute of Biometry and Clinical Epidemiology, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyExperimental and Clinical Research Center, a Cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité—Universitätsmedizin Berlin, Berlin, GermanyExperimental and Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyMax-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, GermanyDepartment of Neurology, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, GermanyNeuroscience Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, GermanyExperimental and Clinical Research Center, a Cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité—Universitätsmedizin Berlin, Berlin, GermanyExperimental and Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, GermanyMax-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, GermanyNeuroscience Clinical Research Center, Charité—Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, GermanyBackgroundThis 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.MethodsThe 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).DiscussionThe 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.https://www.frontiersin.org/articles/10.3389/fneur.2023.1258635/fullmultiple sclerosisdisease progressionoutcome measuresgaitbalancemotor performance |
| spellingShingle | Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Eva-Maria Dorsch Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Hanna Marie Röhling Dario Zocholl Lorena Hafermann Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Friedemann Paul Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Tanja Schmitz-Hübsch Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study Frontiers in Neurology multiple sclerosis disease progression outcome measures gait balance motor performance |
| title | Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study |
| title_full | Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study |
| title_fullStr | Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study |
| title_full_unstemmed | Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study |
| title_short | Progression events defined by home-based assessment of motor function in multiple sclerosis: protocol of a prospective study |
| title_sort | progression events defined by home based assessment of motor function in multiple sclerosis protocol of a prospective study |
| topic | multiple sclerosis disease progression outcome measures gait balance motor performance |
| url | https://www.frontiersin.org/articles/10.3389/fneur.2023.1258635/full |
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