Relationship between growth and ambulation loss in Duchenne muscular dystrophy boys on steroids.

ABSTRACT:

Background and purpose

Treatment with glucocorticoids (GCs) is part of the standard of care in Duchenne muscular dystrophy, but excess weight gain and height stunting are common side-effects. It is still unclear how these growth-related side-effects affect motor function.

Methods

This retrospective cohort study utilized 2228 observations from 648 participants in the UK NorthStar database who had growth and ambulation data recorded between 2006 and 2020. Joint modelling was used to analyse the effect of longitudinal growth centiles on loss of ambulation with respect to GC type and regimen.

Results

Loss of ambulation was observed in 113 patients. National estimates of loss of ambulation age were updated by GC group and showed no significant association between loss of a

SUBMITTER: Stimpson G 

PROVIDER: S-EPMC11555026 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature