Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non-ambulatory males with Duchenne muscular dystrophy from MD STARnet.
Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non-ambulatory males with Duchenne muscular dystrophy from MD STARnet.
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来自MD STARnet的持续糖皮质激素治疗对杜氏肌营养不良症卧床男性患者心肺功能影响的评价
作者：
皮质类固醇已被证明可以改善Duchenne肌营养不良症(DMD)的肌肉力量和延缓行走能力丧失(LOA)，并被认为是标准护理，尽管有显著的副作用。这项研究的目的是评估LOA后的皮质类固醇治疗是否对DMD男孩的心或肺功能有利。我们使用肌营养不良症监测、跟踪和研究网络(MD STARnet)在398名患有DMD的非卧床男孩中表征了皮质类固醇的使用与左心功能异常或预计用力肺活量(PpFVC)异常的发病之间的关系。用Kaplan-Meier曲线估计皮质类固醇使用组的发病时间；用COX比例风险模型估计危险比(HR)、S及相应的95%可信区间。我们发现不同的皮质类固醇使用组在左心功能异常发作的时间上没有差异。我们观察到，在LOA后1年接受皮质类固醇≥治疗的男孩，与未使用皮质类固醇或在LOA后1年内停止使用皮质类固醇的男孩相比，从LOA到首次异常PFVC的时间更长。我们的研究结果表明，除LOA外，皮质类固醇的使用与左心功能异常的发病无关，但与异常ppFVC的发病延迟显著相关。对失去行走能力的DMD男孩使用皮质类固醇的前瞻性研究可能会确定益处，并可以更好地阐明风险，从而允许更有效地咨询LOA后继续治疗的患者。
Corticosteroids have been shown to improve muscle strength and delay loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) and are considered standard of care despite significant side-effects. The objective of this study is to evaluate whether corticosteroid treatment after LOA is beneficial for cardiac or pulmonary functions among boys with DMD. We used the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to characterize associations between corticosteroid use and onset of abnormal left ventricular (LV) function or abnormal percent predicted forced vital capacity (ppFVC) among 398 non-ambulatory boys with DMD. Kaplan-Meier curve estimation was used to compare time to onset by corticosteroid use groups; Cox proportional hazards modeling was used to estimate hazards ratios (HR)s and corresponding 95% confidence intervals. We found no differences in time to onset of abnormal LV function by corticosteroid use groups. We observed a longer time from LOA to first abnormal ppFVC in boys that were treated with corticosteroid ≥1 year beyond LOA compared to those with no corticosteroid use or those who stopped corticosteroid use within 1 year of LOA. Our findings show no association of corticosteroid use beyond LOA with the onset of abnormal LV function, but a significant association with a delay in onset of abnormal ppFVC. Prospective studies of corticosteroid use in boys with DMD who have lost ambulation may identify benefits and can better elucidate risks, allowing for more effective counseling of patients on continuing treatment after LOA.
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影响因子：
2.8
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