Growth, bone health & ambulatory status of boys with DMD treated with daily vs. intermittent oral glucocorticoid regimen
Growth, bone health & ambulatory status of boys with DMD treated with daily vs. intermittent oral glucocorticoid regimen
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DOI：
10.1016/j.bone.2018.07.019
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发表时间：
2018-11-01
期刊：
影响因子：
4.1
通讯作者：
Mughal, M. Z.
中科院分区：
文献类型：
--
作者：
Crabtree, N. J.;Adams, J. E.;Mughal, M. Z.
口服糖皮质激素（GC）可保持男孩杜氏肌营养不良症（DMD）的肌肉力量并延长行走时间。虽然在男孩服用GC中有椎体骨折的报道，但不同GC方案的骨折率尚未调查。这项实用的纵向研究的目的是比较每日（daily）或间歇（间歇性）口服GC方案的DMD男孩的生长、体重、骨密度（BMD）、椎体骨折（VF）和活动状态。来自两个中心的50名DMD男孩的方便样本被纳入研究；25名男孩分别接受每日或间歇治疗。分析3个时间点腰椎骨密度（LS BMAD）、DXA测量的全身减脑骨密度（TBLH）和pQCT测量的前臂远端骨密度、GC暴露、VF评估和运动状态；基线，1年和2年。在基线时，年龄、GC持续时间或任何骨骼参数没有差异。然而，DAILY组男孩较矮(身高SDS DAILY = -1.4(0.9)；间歇性= -0.8(1.0),p = 0.04)， BMI较高(BMI SDS DAILY = 1.5(0.9)；间歇= 0.8(1.0),p = 0.01)。2年后，DAILY男孩逐渐变矮(δ身高SDS DAILY = -0.9(1.1)；间歇= +0.1(0.6),p < 0.001)。在他们的2年评估中，5名DAILY和10名间歇性男孩不能走动。每日男孩比间歇男孩有更多的VFs （10 vs 2; chi(2) p = 0.008）。各组间BMAD SDS保持不变。TBLH和桡骨骨密度明显下降，但损失速率无显著差异。总之，有一种趋势是，更多的男孩每天服用GCs，但代价是更多的VFs，更大的肥胖和显著减少的增长。相比之下，接受间歇性GCs治疗的男孩较少发生椎体骨折，但有更多男孩失去独立行走能力的趋势。
Oral glucocorticoids (GC) preserve muscle strength and prolong walking in boys with Duchenne muscular dystrophy (DMD). Although vertebral fractures have been reported in boys taking GC, fracture rates for different GC regimes have not been investigated. The aim of this pragmatic longitudinal study was to compare growth, body mass, bone mineral density (BMD), vertebral fractures (VF) and ambulatory status in boys with DMD on daily (DAILY) or intermittent (INTERMITTENT), oral GC regimens.A convenience sample of 50 DMD boys from two centres was included in the study; 25 boys each were on the DAILY or INTERMITTENT regimen. Size adjusted lumbar spine BMD (LS BMAD), total body less head BMD (TBLH), by DXA and distal forearm bone densities by pQCT, GC exposure, VF assessment and ambulatory status were analysed at three time points; baseline, 1 and 2 years.At baseline, there were no differences in age, GC duration or any bone parameters. However, DAILY boys were shorter (height SDS DAILY = -1.4(0.9); INTERMITTENT = -0.8(1.0), p = 0.04) with higher BMI (BMI SDS DAILY = 1.5(0.9); INTERMITTENT = 0.8(1.0), p = 0.01). Over 2 years, DAILY boys got progressively shorter (delta height SDS DAILY = -0.9(1.1); INTERMITTENT = +0.1(0.6), p < 0.001). At their 2 year assessment, 5 DAILY and 10 INTERMITTENT boys were non-ambulant. DAILY boys had more VFs than INTERMITTENT boys (10 versus 2; chi(2) p = 0.008). BMAD SDS remained unchanged between groups. TBLH and radius BMD declined significantly but the rate of loss was not different.In conclusion, there was a trend for more boys on daily GCs to remain ambulant but at the cost of more VFs, greater adiposity and markedly diminished growth. In contrast, boys on intermittent GCs had fewer vertebral fractures but there was a trend for more boys to loose independent ambulation.