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Lookup NU author(s): Dr Marianela SchiavaORCiD, Dr Claire WoodORCiD, Robert Muni Lofra, Dr Anna Mayhew, Professor Michela GuglieriORCiD, Emerita Professor Elaine McCollORCiD, Emerita Professor Katherine Bushby, Professor Volker StraubORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2025 The Author(s). Developmental Medicine & Child Neurology published by John Wiley & Sons Ltd on behalf of Mac Keith Press.Aim: To examine the factors influencing height, weight, and body mass index (BMI) z-scores, and the relationship between them and motor performance, in boys with Duchenne muscular dystrophy (DMD). Method: This was a randomized, double-blind, parallel group trial involving 32 study sites across five countries. Height, weight, BMI z-scores, and clinical outcome assessments (COAs)—rise from supine velocity, 10-m walk/run velocity, NorthStar Ambulatory Assessment, and 6-minute walk test—were analysed in 4-year-old to 7-year-old boys with DMD randomized to 0.75 mg/kg/day prednisone, 0.75 mg/kg/day intermittent prednisone, or 0.90 mg/kg/day deflazacort in the FOR-DMD study. Trajectories were modelled using a linear mixed-effects model and correlations were explored through Spearman's partial correlations. Results: In 194 boys with DMD, higher height at glucocorticoid initiation was associated with slower growth (p < 0.001) and older age was associated with increased weight gain (p = 0.001). Glucocorticoid type and regimen influenced height and weight trajectories but not BMI. Changes in height and weight z-scores were negatively correlated with COAs (p < 0.05 in all cases). Correlations were weak 3 years after glucocorticoid initiation and moderate after 5 years (closer to the age of loss of ambulation). Interpretation: Changes in anthropometric measures after glucocorticoid initiation are associated with COA performance and larger correlations closer to the age of loss of ambulation. This emphasizes the need for weight management strategies and discussions that support treatment.
Author(s): Schiava M, Dang UJ, Wood C, Wong SC, Ward LM, Muni Lofra R, Mayhew A, Martens WB, Gregory S, Griggs RC, Guglieri M, Manzur A, Rachel A, Gangfuss A, Childs AM, Kumar A, Herr BE, Darras BT, Speed C, Campbell C, McDonald CM, Wilichowski E, McColl E, Pegoraro E, Ciafaloni E, Henricson EK, Ricci F, Vita GL, Baranello G, Vita G, Roper H, McMillan HJ, Horrocks IA, Hughes I, Howard JF, Kirschner J, Han JJ, Mah JK, Statland JM, Wilkinson J, Bushby K, O'Reardon K, Flanigan KM, Hart KA, Morrison L, Maggi L, Bello L, von der Hagen M, Brown MW, Thangarajh M, Wicklund M, McDermott MP, Eagle M, Krzesniak-Swinarska M, Kuntz NL, Joyce N, Shieh PB, Kang PB, Tawil R, Barohn RJ, Finkel RS, Butterfield RJ, Spinty S, Chang T, Mongini TE, Willis T, Schara-Schmidt U, Straub V, Burnette WB, King WM, Alhaswani Z
Publication type: Article
Publication status: Published
Journal: Developmental Medicine and Child Neurology
Year: 2025
Pages: epub ahead of print
Online publication date: 31/08/2025
Acceptance date: 27/06/2025
Date deposited: 30/09/2025
ISSN (print): 0012-1622
ISSN (electronic): 1469-8749
Publisher: John Wiley and Sons Inc
URL: https://doi.org/10.1111/dmcn.16437
DOI: 10.1111/dmcn.16437
Data Access Statement: The first author and corresponding author have full access to the data used in the analyses for this manuscript. The first author takes full responsibility for the data, the analyses, interpretation, and the conduct of the research. The first and corresponding author have the right to publish any and all data. De-identified participant data will be archived on the NINDS website for archived clinical research datasets (URL number not available yet) from 08-31-2023.
PubMed id: 40887311
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