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Research paper
Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
  1. Valeria Ricotti1,
  2. Deborah A Ridout2,
  3. Elaine Scott3,
  4. Ros Quinlivan1,4,
  5. Stephanie A Robb1,
  6. Adnan Y Manzur1,
  7. Francesco Muntoni1,4,
  8. on behalf of the NorthStar Clinical Network
  1. 1Dubowitz Neuromuscular Centre, UCL Institute of Child Health and Great Ormond Street Hospital Foundation Trust, London, UK
  2. 2Centre for Paediatric Epidemiology and Biostatistics, UCL Institute of Child Health and Great Ormond Street Hospital Foundation Trust, London, UK
  3. 3Muscular Dystrophy Campaign, Sheffield, UK
  4. 4MRC Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, London, UK
  1. Correspondence to Dr Francesco Muntoni, Dubowitz Neuromuscular Centre, UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK; f.muntoni{at}ucl.ac.uk

Abstract

Objective To assess the current use of glucocorticoids (GCs) in Duchenne muscular dystrophy in the UK, and compare the benefits and the adverse events of daily versus intermittent prednisolone regimens.

Design A prospective longitudinal observational study across 17 neuromuscular centres in the UK of 360 boys aged 3–15 years with confirmed Duchenne muscular dystrophy who were treated with daily or intermittent (10 days on/10 days off) prednisolone for a mean duration of treatment of 4 years.

Results The median loss of ambulation was 12 years in intermittent and 14.5 years in daily treatment; the HR for intermittent treatment was 1.57 (95% CI 0.87 to 2.82). A fitted multilevel model comparing the intermittent and daily regiments for the NorthStar Ambulatory Assessment demonstrated a divergence after 7 years of age, with boys on an intermittent regimen declining faster (p<0.001). Moderate to severe side effects were more commonly reported and observed in the daily regimen, including Cushingoid features, adverse behavioural events and hypertension. Body mass index mean z score was higher in the daily regimen (1.99, 95% CI 1.79 to 2.19) than in the intermittent regimen (1.51, 95% CI 1.27 to 1.75). Height restriction was more severe in the daily regimen (mean z score −1.77, 95% CI −1.79 to −2.19) than in the intermittent regimen (mean z score −0.70, 95% CI −0.90 to −0.49).

Conclusions Our study provides a framework for providing information to patients with Duchenne muscular dystrophy and their families when introducing GC therapy. The study also highlights the importance of collecting longitudinal natural history data on patients treated according to standardised protocols, and clearly identifies the benefits and the side-effect profile of two treatment regimens, which will help with informed choices and implementation of targeted surveillance.

  • Muscular Dystrophy

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