Height, weight, and body mass index trajectories and their correlation with functional outcome assessments in boys with Duchenne muscular dystrophy

Publikation: Beitrag in FachzeitschriftForschungsartikelBeigetragenBegutachtung

Beitragende

  • Newcastle University
  • Carleton University
  • Newcastle upon Tyne Hospitals NHS Foundation Trust
  • University of Glasgow
  • University of Ottawa
  • University of Rochester Medical Center

Abstract

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.

Details

OriginalspracheEnglisch
Seiten (von - bis)429-440
Seitenumfang12
FachzeitschriftDevelopmental Medicine and Child Neurology
Jahrgang68
Ausgabenummer3
Frühes Online-Datum31 Aug. 2025
PublikationsstatusVeröffentlicht - März 2026
Peer-Review-StatusJa

Externe IDs

PubMed 40887311