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首页> 外文期刊>Journal of Neurology, Neurosurgery and Psychiatry >Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
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Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy

机译：间歇性糖皮质激素与每日糖皮质激素治疗对杜氏肌营养不良的男孩的长期益处和不良影响

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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.
机译：目的：评估目前在英国的杜兴氏肌营养不良症中使用糖皮质激素（GCs）的情况，并比较每日泼尼松龙和间歇性泼尼松龙方案的益处和不良事件。设计：一项在英国的17个神经肌肉中心进行的前瞻性纵向观察研究，研究对象是360名3-15岁确诊为Duchenne肌营养不良症的男孩，他们接受每日或间歇性泼尼松龙治疗（间歇治疗10天/ 10天），平均治疗时间4年。结果：间歇性活动下降的中位数为12年，日常治疗为14.5年。间歇治疗的HR为1.57（95％CI为0.87至2.82）。一项适用于NorthStar动态评估的间歇方案和日常方案的多层次拟合模型显示，7岁后男孩出现了差异，间歇方案的男孩下降得更快（p <0.001）。在日常方案中，中度至严重的副作用更为常见，包括库欣类药物特征，不良行为事件和高血压。每日方案（1.99，95％CI 1.79至2.19）的体重指数平均z评分高于间歇方案（1.51，95％CI 1.27至1.75）。日常方案中的身高限制（z平均值-1.77，95％CI -1.79至-2.19）比间歇方案（z平均值-0.70，95％CI -0.90至-0.49）更严格。结论：我们的研究提供了一个框架，可为采用GC治疗的杜氏肌营养不良患者及其家属提供信息。这项研究还强调了收集按照标准化方案治疗的患者的纵向自然历史数据的重要性，并清楚地确定了两种治疗方案的益处和副作用，这将有助于明智的选择和实施有针对性的监测。

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