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Observational Study
. 2025 Jul 16;96(8):794-801.
doi: 10.1136/jnnp-2024-335223.

Observational study of changes to glucocorticosteroid prescribing patterns in duchenne muscular dystrophy in the UK over the last decade

Gregory Landon  1   2 , Georgia Stimpson  2 , Michela Guglieri  3 , Anna Sarkozy  2   4 , Adnan Y Manzur  1   2 ; UK NorthStar Clinical Network; Francesco Muntoni  5   2 , Giovanni Baranello  1   2
Collaborators, Affiliations
Observational Study

Observational study of changes to glucocorticosteroid prescribing patterns in duchenne muscular dystrophy in the UK over the last decade

Gregory Landon et al. J Neurol Neurosurg Psychiatry. .

Abstract

Background: Glucocorticosteroids (GC) are standard-of-care treatment for most boys with duchenne muscular dystrophy (DMD). GC use has changed over time with evolving evidence, and we describe GC patterns, dosing and side-effects in the UK over 11 years.

Method: NorthStar data from 2012 to 2022 were analysed to understand GC type, regime and starting age. GC dose with age, patterns of GC switching and side-effect profiles by type and regime were also analysed. Participants attributed to 'other' regimes were queried and details were included.

Results: Data on GC usage were available for 1117 boys, across 6905 observations, with 74% of boys GC treated. Prednisolone was the most common regime in the period (65% of assessments) but deflazacort prescription has increased (17% in 2012 and 43% in 2022). Daily regimes were more common (66% of assessments), and the incidence of intermittent (10 days on/10 days off) regimes has declined (46% in 2012 and 26% in 2022). Older participants were more commonly on less than recommended doses, and this was more common in those on deflazacort or daily regimes. Gastrointestinal symptoms and cushingoid features were more common in those on deflazacort than prednisolone, while increased appetite, cushingoid features, gastrointestinal symptoms and insomnia were more common in those on daily than intermittent regimes.

Conclusions: The use of deflazacort and daily regimes has steadily increased across the UK North Star Network in the last decade. This study provides one of the largest up-to-date real-world set of data of evolution in prescription patterns and the occurrence of side-effects in different groups of GC-treated DMD.

Keywords: DYSTROPHIN; MUSCULAR DYSTROPHY; NEUROMUSCULAR; STEROIDS.

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Conflict of interest statement

Competing interests: MG has participated in advisory boards for PTC Therapeutics, Capricor, Pfizer and NS Pharma. She has research collaborations with ReveraGen, she is or has been Principal Investigator for clinical trials with Roche, Italfarmaco, Santhera, ReveraGen, Summit, Pfizer, PTC Therapeutics; FM reports participation to 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; GB is 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 (AveXis), and Roche and has worked as principal investigator of SMA studies sponsored by Novartis Gene Therapies, and Roche; GL, GS, AYM and AS have no conflict of interest to declare.

Figures

Figure 1
Figure 1. Data cleaning and summary of GC data completeness. GC, glucocorticosteroids.
Figure 2
Figure 2. Changes in (A) type and (B) regime of GC prescribed over time. GC, glucocorticosteroids.
Figure 3
Figure 3. Hydrocortisone equivalent dose of GC prescribed by age and GC regime. GC, glucocorticosteroids.
Figure 4
Figure 4. Trends in GC start age and time to treat by year. GC, glucocorticosteroids.
See this image and copyright information in PMC

References

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