Dubowitz Neuromuscular Centre, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.
Population, Policy and Practice Department, Faculty of Population Health Sciences, UCL Great Ormond Street Institute of Child Health, London, UK.
Eur J Neurol. 2024 Dec;31(12):e16415. doi: 10.1111/ene.16415. Epub 2024 Sep 9.
Treatment with glucocorticoids (GCs) is part of the standard of care in Duchenne muscular dystrophy, but excess weight gain and height stunting are common side-effects. It is still unclear how these growth-related side-effects affect motor function.
This retrospective cohort study utilized 2228 observations from 648 participants in the UK NorthStar database who had growth and ambulation data recorded between 2006 and 2020. Joint modelling was used to analyse the effect of longitudinal growth centiles on loss of ambulation with respect to GC type and regimen.
Loss of ambulation was observed in 113 patients. National estimates of loss of ambulation age were updated by GC group and showed no significant association between loss of ambulation risk and absolute growth centile. However, yearly drift in weight and/or height centile had an associated risk effect on loss of ambulation. Over a 2-year period, a yearly drift in weight from the 50th to the 75th, 75th to the 90th and 90th to the 95th centile was associated with 138%, 118% and 64% increased risk of loss of ambulation, respectively. Conversely, a 2-year drift in height from the 50th to the 25th, 25th to the 10th and 10th to the 5th centile was associated with 53%, 49% and 35% decreased risk of loss of ambulation, respectively.
Our results suggest a complex relationship between growth and loss of ambulation in Duchenne muscular dystrophy boys on chronic GCs, the first step in understanding the effects of drugs which also affect growth patterns.
在杜氏肌营养不良症的标准治疗中，糖皮质激素（GCs）的治疗是其中的一部分，但体重过度增加和身高发育迟缓是常见的副作用。目前尚不清楚这些与生长相关的副作用如何影响运动功能。
本回顾性队列研究利用了英国 NorthStar 数据库中 648 名参与者的 2228 次观察结果，这些参与者在 2006 年至 2020 年期间记录了生长和步行数据。联合建模用于分析纵向生长百分位数对 GC 类型和方案对步行丧失的影响。
113 名患者出现了步行丧失。根据 GC 组更新了全国步行丧失年龄的估计值，结果显示步行丧失风险与绝对生长百分位数之间没有显著关联。然而，体重和/或身高百分位的逐年变化与步行丧失的风险有关。在 2 年期间，体重从第 50 百分位到第 75 百分位、第 75 百分位到第 90 百分位和第 90 百分位到第 95 百分位的每年变化分别与步行丧失风险增加 138%、118%和 64%相关。相反，身高从第 50 百分位到第 25 百分位、第 25 百分位到第 10 百分位和第 10 百分位到第 5 百分位的每年变化分别与步行丧失风险降低 53%、49%和 35%相关。
我们的结果表明，在慢性接受 GCs 治疗的杜氏肌营养不良症男孩中，生长与步行丧失之间存在复杂的关系，这是了解也影响生长模式的药物影响的第一步。
