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. 2025 Nov;32(11):e70414.
doi: 10.1111/ene.70414.

Joint Modelling of Growth and Motor Function Centiles in Corticosteroids Treated Boys With Duchenne Muscular Dystrophy

Georgia Stimpson  1 , Deborah Ridout  2 , Amy Wolfe  1   3 , Evelin Milev  1   3 , Emer O'Reilly  1   3 , Adnan Manzur  1 , Anna Sarkozy  1 , Francesco Muntoni  1   3 , Giovanni Baranello  1   3 ; UK NorthStar Clinical Network
Collaborators, Affiliations

Joint Modelling of Growth and Motor Function Centiles in Corticosteroids Treated Boys With Duchenne Muscular Dystrophy

Georgia Stimpson et al. Eur J Neurol. 2025 Nov.

Abstract

Background: Corticosteroid (CS) treated boys with DMD display higher rates of height stunting, higher weight gain, improved motor function scores and delayed loss of ambulation compared to untreated patients. However, the relationship between growth and motor function has historically been understudied due to modelling complexities.

Methods: In this analysis, we use the newly developed motor function centiles for the NSAA, RFF and 10MWR. We consider each combination of growth (height and weight SD) and motor function using multivariate regression models controlling for differential CS treatment (prednisolone/deflazacort, daily/intermittent). This allows inference on the growth and motor function outcomes separately and on the relationship between the outcomes.

Results: We consider 559 steroid-treated boys with DMD between the ages of 5 and 16 over 1643 assessments. Better motor function trajectories were observed in those treated with daily CS, with the deflazacort daily group displaying a positive NSAA centile trajectory (annual change of 0.07 SD). There was a mild, negative pairwise correlation between the annual changes in NSAA and 10MWR Z-Scores, and height and weight Z-Scores, ranging from -0.25 to -0.36. This indicated that patients with a milder weight gain or more severe height stunting trajectory with respect to their CS treatment were more likely to exhibit a more favourable NSAA or 10MWR trajectory over time.

Conclusions: This work describes the complex relationships between motor function, CS treatment and growth and provides insights for conversations about the relative benefits and negative effects of CS.

Keywords: 10 meter walk run; Duchenne muscular dystrophy; NorthStar ambulatory assessment; motor function; rise from floor.

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Conflict of interest statement

F.M. reports participation in Scientific Advisory boards and teaching initiatives for Novartis, Biogen, Roche; he is involved as an investigator in clinical trials from Novartis, Biogen, and Roche. Both institutions (UCL and GOSH) receive funding from Biogen and Roche for the SMAREACH SMA registry. G.B. is the PI of clinical trials by Pfizer, NS Pharma, and Reveragen, and has received speaker and/or consulting fees from Sarepta, PTC Therapeutics, Biogen, Novartis Gene Therapies Inc. (AveXis), and Roche and has worked as principal investigator of SMA studies sponsored by Novartis Gene Therapies Inc., and Roche. A.W. has received speaker or consulting fees from Biogen and Novartis Gene Therapies, and is involved as an investigator in clinical trials from Novartis, Biogen, and Roche. EOR reports participation in an advisory board for Roche. E.M. provides consultancy services through ATOM International Ltd. for the following companies: Amicus Therapeutics Pty Ltd., Genethon, Italfarmaco, NS Pharma, Pfizer, Edgewise Therapeutics, Sarepta, Dyne Therapeutics. G.S., D.R., A.M. and A.S. have no conflicts of interest to declare.

Figures

FIGURE 1
FIGURE 1
Data cleaning process.
FIGURE 2
FIGURE 2
Example growth and NSAA trajectories and comparative Z‐Score trajectories for 5 patients treated with prednisolone daily, who display varying NSAA trajectories (slopes of the 10th, 30th, 50th, 70th and 90th centiles).
FIGURE 3
FIGURE 3
Marginal correlations of motor function and growth with age.
See this image and copyright information in PMC

References

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