Landon Gregory, Stimpson Georgia, Guglieri Michela, Sarkozy Anna, Manzur Adnan Y, Muntoni Francesco, Baranello Giovanni
Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Dubowitz Neuromuscular Centre, UCL GOS Institute of Child Health, London, UK.
J Neurol Neurosurg Psychiatry. 2025 Jul 16;96(8):794-801. doi: 10.1136/jnnp-2024-335223.
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.
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.
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.
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.
糖皮质激素（GC）是大多数杜氏肌营养不良症（DMD）男孩的标准治疗方法。随着证据的不断发展，GC的使用也发生了变化，我们描述了英国11年来GC的使用模式、剂量和副作用。
分析2012年至2022年的北极星数据，以了解GC的类型、治疗方案和起始年龄。还分析了GC剂量随年龄的变化、GC转换模式以及不同类型和治疗方案的副作用情况。对归类为“其他”治疗方案的参与者进行了询问并纳入了详细信息。
有1117名男孩的GC使用数据，共6905次观察，其中74%的男孩接受了GC治疗。泼尼松龙是该时期最常见的治疗方案（65%的评估），但地夫可特的处方量有所增加（2012年为17%，2022年为43%）。每日治疗方案更为常见（66%的评估），间歇性（10天用药/10天停药）治疗方案的发生率有所下降（2012年为46%，2022年为26%）。年龄较大的参与者更常使用低于推荐剂量的药物，这在使用地夫可特或每日治疗方案的患者中更为常见。与泼尼松龙相比，地夫可特使用者出现胃肠道症状和库欣样特征更为常见，而每日治疗方案的使用者比间歇性治疗方案的使用者食欲增加、库欣样特征、胃肠道症状和失眠更为常见。
在过去十年中，英国北极星网络中地夫可特和每日治疗方案的使用稳步增加。本研究提供了最大的最新真实世界数据集之一，用于了解不同组接受GC治疗的DMD患者处方模式的演变和副作用的发生情况。
