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 |
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| Authors: | 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; Deconinck N; Tulinius M; Flanigan K; Henricson E; Dutra de Resende MB; Vita GL; Schara U; Kirschner J; Topaloglu H; Monges S; Cances C; Domingos J; Ricotti V; Selby V; Wolfe A; Abbott L; Milev E; Panagiotopoulou E; Iodice M; Ash M; Voit T; Decostre V; Gilabert S; Hogrel J-Y; Murphy A; Mayhew A; Van der Holst M; Krom YD; van Heur-Neuman MJ; Jansen M; Pelsma M; Bobbert M; Verschuuren JJGM; Robb S; Quinlivan R; Sarkozy A; Munot P; Baranello G; Scoto M; Main M; Patel H; Samsuddin S; Gupta VA; Bushby K; Bertolli C; Muni-Lofra R; James M; Moat D; Sodhi J; Roper H; Parasuraman D; McMurchie H; Rabb R; Pysden K; Pallant L; Peachey G; Madhu R; Shillington A; Jungbluth H; Sheehan J; Spahr R; Bateman E; Cammiss C; Groves L; Emery N; Baxter P; Goulborne N; Senior M; Scott E; Hartley L; Parsons B; Mason F; Jenkins L; Toms B; Frimpong-Ansah C; Jarvis H; Dalgleish J; Keddie A; Di Marco M; Dunne J; Miah A; Selley A; Geary M; Palmer J; Greenfield K; MacAuley S; Robbins H; Iqbal M; Ward C; Taylor J; O'Hara A; Tewnion J; Chandratre S; Ramdas S; White M; Ramjattan H; Yirrel J; Arora H; Willcocks RJ; Lott DJ; Senesac CR; Triplett WT; Byrne BJ; Walte GA; Sweeney HL; Harrington AT; Tennekoon GI; Zilke KL; Finanger EL; Russman BS; Daniels MJ; Xu D; Finkel RS; Bettica P; Billeter M; Van den Hauwe M; Rybalsky I; Shellenbarger KC; McCormick AE; McGuire MN; Bonarrigo K; Fowler AE; Kiefer M; Bange J; Hu S; Chik F; Chen J; Shen P |
| Source: | PLoS ONE, July 2024 |
| Publisher Information: | Public Library of Science |
| Publication Year: | 2024 |
| Collection: | Newcastle University Library ePrints Service |
| Description: | © 2024 Muntoni et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.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 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. |
| Document Type: | article in journal/newspaper |
| Language: | unknown |
| Relation: | https://eprints.ncl.ac.uk/299847 |
| Availability: | https://eprints.ncl.ac.uk/299847 |
| Rights: | https://creativecommons.org/licenses/by/4.0/ |
| Accession Number: | edsbas.DD79C281 |
| Database: | BASE |