ArticleEuropean journal of neurology2024
Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.
Article in European journal of neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 2 of them syntheses that pooled it.
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Who cites it
12 citing papers in PubMed, 2 syntheses or guidelines pooled it, 13 citations in OpenAlex.
- Improving survival in Duchenne muscular dystrophy across eras: a systematic review and cumulative meta-analysis.Journal of neurology · 2026Pooled it
- Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis.Frontiers in neurology · 2024Pooled it
- Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol.European journal of neurology · 2025Trial
- The Effect of Corticosteroids on Pulmonary Function in a Large Cohort of Late Adolescents and Adults With Duchenne Muscular Dystrophy.Muscle & nerve · 2026Observational
- A high-fat, high-sucrose diet exacerbates muscle and metabolic pathology and undermines glucocorticoid efficacy in dystrophin-deficient mice.The Journal of physiology · 2026Article
- Phospho-mimic βIII-tubulin rescues microtubule and cardiac defects in Duchenne muscular dystrophy mice.Journal of molecular and cellular cardiology · 2026Article
- Respiratory function in Becker muscular dystrophy: a comprehensive longitudinal study.Journal of neurology, neurosurgery, and psychiatry · 2026Article
- Telemedicine and remote monitoring in neuromuscular diseases: Challenges and opportunities.Journal of neuromuscular diseases · 2026Review
- The Importance of Early Diagnosis and Treatment to Limit the Impact of Dystrophic Cardiomyopathy-We've Seen This Film Before, and We Didn't Like the Ending.Journal of cardiovascular development and disease · 2025Review
- Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines.Journal of neurology · 2025Article
- The effects of glucocorticoids on cardiac function of patients with Duchenne muscular dystrophy: benefit or not?European journal of pediatrics · 2025Review
- Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.European journal of neurology · 2024Article
Corrections and comments
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Authors and funding
23 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
background and purposeThe transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late-stage clinical outcomes.
methodsThis was a retrospective single-centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records.
resultsIn all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow-up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status.
conclusionGlucocorticoids after LOA preserve late-stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.
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