Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy.
Mayhew, Anna G Moat, Dionne McDermott, Michael P Eagle, Michelle Griggs, Robert C King, Wendy M James, Meredith K Muni-Lofra, Robert Shillington, Alison Gregson, Sarah
Mayhew, Anna G
Moat, Dionne
McDermott, Michael P
Eagle, Michelle
Griggs, Robert C
King, Wendy M
James, Meredith K
Muni-Lofra, Robert
Shillington, Alison
Gregson, Sarah
Abstract
The purpose of this study was to quantitate motor performance in 196 genetically confirmed steroid-naïve boys with Duchenne muscular dystrophy (DMD), to evaluate the test-retest reliability of measures of motor performance in young DMD boys, and to assess correlations among the different functional outcomes including timed tests. Boys aged 4-7 years were recruited in the FOR-DMD study, a comparative effectiveness study of different steroid regimens in DMD. Eligible boys had to be able to rise from the floor independently and to perform pulmonary function testing consistently. The boys were evaluated with standardized assessments at the screening and baseline visits at 32 sites in 5 countries (US, UK, Canada, Italy, Germany). Assessments included timed rise from floor, timed 10 m walk/run, six-minute walk distance, North Star Ambulatory Assessment (NSAA) and forced vital capacity (FVC). Mean age at baseline was 5.9 years (range 4.1-8.1 years). Test-retest reliability was high for functional assessments, regardless of time lag between assessments (up to 90 days) and for the majority of age groups. Correlations were strong among the functional measures and timed tests, less so with FVC. Physiotherapy measures are reliable in a young, steroid-naïve population and rise from floor velocity appears to be a sensitive measure of strength in this population.
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Date
2022-02-26
Type
Article
Subject
Paediatrics
Collections
Citation
Mayhew AG, Moat D, McDermott MP, Eagle M, Griggs RC, King WM, James MK, Muni-Lofra R, Shillington A, Gregson S, Pallant L, Skura C, Staudt LA, Eichinger K, McMurchie H, Rabb R, Di Marco M, Brown S, Zanin R, Arnoldi MT, McIntyre M, Wilson A, Alfano LN, Lowes LP, Blomgren C, Milev E, Iodice M, Pasternak A, Chiu A, Lehnert I, Claus N, Dieruf KA, Rolle E, Nicorici A, Andres B, Hobbiebrunken E, Roetmann G, Kern V, Civitello M, Vogt S, Hayes MJ, Scholtes C, Lacroix C, Gunn T, Warner S, Newman J, Barp A, Kundrat K, Kovelman S, Powers PJ, Guglieri M; Muscle Study Group and TREAT-NMD. Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2022 Jun;32(6):460-467. doi: 10.1016/j.nmd.2022.02.012. Epub 2022 Feb 26
Journal / Source Title
Neuromuscular Disorders
DOI
10.1016/j.nmd.2022.02.012
PMID
35618576
Publisher
Pergamon Press
Publisher’s URL
https://www.sciencedirect.com/journal/neuromuscular-disorders