Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study
Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the ‘meaningfulness’ of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function...
| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , |
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| Format: | Journal article |
| Language: | English |
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Public Library of Science
2024
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| _version_ | 1811140038564511744 |
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| author | Muntoni, F Signorovitch, J Sajeev, G Done, N Yao, Z Goemans, N McDonald, C Mercuri, E Niks, EH Wong, B Vandenborne, K Straub, V de Groot, IJM Tian, C Manzur, A Dieye, I Lane, H Ward, SJ Servais, L PRO-DMD-01 study investigators Association Française contre les Myopathies The UK NorthStar Clinical Network ImagingDMD investigators cTAP |
| author_facet | Muntoni, F Signorovitch, J Sajeev, G Done, N Yao, Z Goemans, N McDonald, C Mercuri, E Niks, EH Wong, B Vandenborne, K Straub, V de Groot, IJM Tian, C Manzur, A Dieye, I Lane, H Ward, SJ Servais, L PRO-DMD-01 study investigators Association Française contre les Myopathies The UK NorthStar Clinical Network ImagingDMD investigators cTAP |
| author_sort | Muntoni, F |
| collection | OXFORD |
| description | Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the ‘meaningfulness’ of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function measures in ambulatory DMD, i.e., the minimal degree of measured change needed to be confident that true underlying change has occurred rather than transient variation or measurement error. MDC estimates were compared across multiple data sources, representing >1000 DMD patients in clinical trials and real-world clinical practice settings. Included patients were ambulatory, aged ≥4 to <18 years and receiving steroids. Minimal clinically important differences (MCIDs) for worsening were also estimated. Estimated MDC thresholds for >80% confidence in true change were 2.8 units for the North Star Ambulatory Assessment (NSAA) total score, 1.3 seconds for the 4-stair climb (4SC) completion time, 0.36 stairs/second for 4SC velocity and 36.3 meters for the 6-minute walk distance (6MWD). MDC estimates were similar across clinical trial and real-world data sources, and tended to be slightly larger than MCIDs for these measures. The identified thresholds can be used to inform endpoint definitions, or as benchmarks for monitoring individual changes in motor function in ambulatory DMD. |
| first_indexed | 2024-09-25T04:15:38Z |
| format | Journal article |
| id | oxford-uuid:81da102c-9bc0-4ccc-90fa-13ddcab91cbb |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-09-25T04:15:38Z |
| publishDate | 2024 |
| publisher | Public Library of Science |
| record_format | dspace |
| spelling | oxford-uuid:81da102c-9bc0-4ccc-90fa-13ddcab91cbb2024-07-18T20:03:49ZMeaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center studyJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:81da102c-9bc0-4ccc-90fa-13ddcab91cbbEnglishJisc Publications RouterPublic Library of Science2024Muntoni, FSignorovitch, JSajeev, GDone, NYao, ZGoemans, NMcDonald, CMercuri, ENiks, EHWong, BVandenborne, KStraub, Vde Groot, IJMTian, CManzur, ADieye, ILane, HWard, SJServais, LPRO-DMD-01 study investigatorsAssociation Française contre les MyopathiesThe UK NorthStar Clinical NetworkImagingDMD investigatorscTAPEvaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the ‘meaningfulness’ of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function measures in ambulatory DMD, i.e., the minimal degree of measured change needed to be confident that true underlying change has occurred rather than transient variation or measurement error. MDC estimates were compared across multiple data sources, representing >1000 DMD patients in clinical trials and real-world clinical practice settings. Included patients were ambulatory, aged ≥4 to <18 years and receiving steroids. Minimal clinically important differences (MCIDs) for worsening were also estimated. Estimated MDC thresholds for >80% confidence in true change were 2.8 units for the North Star Ambulatory Assessment (NSAA) total score, 1.3 seconds for the 4-stair climb (4SC) completion time, 0.36 stairs/second for 4SC velocity and 36.3 meters for the 6-minute walk distance (6MWD). MDC estimates were similar across clinical trial and real-world data sources, and tended to be slightly larger than MCIDs for these measures. The identified thresholds can be used to inform endpoint definitions, or as benchmarks for monitoring individual changes in motor function in ambulatory DMD. |
| spellingShingle | Muntoni, F Signorovitch, J Sajeev, G Done, N Yao, Z Goemans, N McDonald, C Mercuri, E Niks, EH Wong, B Vandenborne, K Straub, V de Groot, IJM Tian, C Manzur, A Dieye, I Lane, H Ward, SJ Servais, L PRO-DMD-01 study investigators Association Française contre les Myopathies The UK NorthStar Clinical Network ImagingDMD investigators cTAP Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title | Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title_full | Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title_fullStr | Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title_full_unstemmed | Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title_short | Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study |
| title_sort | meaningful changes in motor function in duchenne muscular dystrophy dmd a multi center study |
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