Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy.
Prophylactic use of cardiac medications for delay of left ventricular dysfunction in Duchenne muscular dystrophy.
复制标题
预防性使用心脏药物来延迟杜氏肌营养不良症的左心室功能障碍。
DOI：
10.1002/bdr2.2260
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发表时间：
2024
影响因子：
2.1
通讯作者：
MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)
中科院分区：
文献类型：
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作者：
Conway,KristinM;Thomas,Shiny;Ciafaloni,Emma;Khan,RabiaS;Mann,JoshuaR;Romitti,PaulA;Mathews,KatherineD;MuscularDystrophySurveillance,TrackingandResearchNetwork(MDSTARnet)
背景Duchenne型肌营养不良症患者左心室功能障碍（LVD）的预防性治疗流行病学支持有限。我们使用回顾性的，基于人群的监测数据，从肌营养不良监测，跟踪和研究网络，以评估是否预防延迟LVD onset.MethodsWe分析了455名男性出生于1982年至2009年。首次超声心动图异常时的年龄（射血分数<55%或短轴缩短分数<28%）决定了LVD的发生。预防定义为LVD前至少1年使用心脏药物。还对皮质类固醇的使用进行了编码。Kaplan-Meier曲线估计和考克斯比例风险模型随时间变化的协变量描述association.ResultsLVD被确定为40.7%，平均发病年龄为14.2岁。20.2%确定了预防措施，57.4%确定了皮质类固醇。与未治疗组相比，预防组LVD发作延迟（p<0.001），功能障碍风险较低（校正风险比[aHR] = 0.39，95%CL = 0.22，0.65）。与未治疗相比，仅连续使用皮质类固醇（aHR = 1.01，95%CL = 0.66，1.53）和仅预防（aHR = 0.67，95%CL = 0.25，1.50）对心脏无保护作用，但预防性治疗加连续使用皮质类固醇可降低功能障碍的风险（aHR = 0.37，95%CL = 0.15，0.80）。结论积极的心脏治疗和监测是Duchenne型肌营养不良症治疗的关键。与临床护理指南一致，本研究支持在记录的LVD之前开始的心脏药物的临床获益，并提示与皮质类固醇联合使用时的进一步获益。
BackgroundEpidemiological support for prophylactic treatment of left ventricular dysfunction (LVD) in Duchenne muscular dystrophy is limited. We used retrospective, population‐based surveillance data from the Muscular Dystrophy Surveillance, Tracking and Research Network to evaluate whether prophylaxis delays LVD onset.MethodsWe analyzed 455 males born during 1982–2009. Age at first abnormal echocardiogram (ejection fraction <55% or shortening fraction <28%) determined LVD onset. Prophylaxis was defined as cardiac medication use at least 1 year prior to LVD. Corticosteroid use was also coded. Kaplan–Meier curve estimation and Cox Proportional Hazard modeling with time‐varying covariates describe associations.ResultsLVD was identified among 40.7%; average onset age was 14.2 years. Prophylaxis was identified for 20.2% and corticosteroids for 57.4%. Prophylaxis showed delayed LVD onset (p< .001) and lower hazard of dysfunction (adjusted hazard ratio [aHR] = 0.39, 95%CL = 0.22, 0.65) compared to untreated. Compared to no treatment, continuous corticosteroids only (aHR = 1.01, 95%CL = 0.66, 1.53) and prophylaxis only (aHR = 0.67, 95%CL = 0.25, 1.50) were not cardioprotective, but prophylaxis plus continuous corticosteroids were associated with lower hazard of dysfunction (aHR = 0.37, 95%CL = 0.15, 0.80).ConclusionsProactive cardiac treatment and monitoring are critical aspects of managing Duchenne muscular dystrophy. Consistent with clinical care guidelines, this study supports clinical benefit from cardiac medications initiated prior to documented LVD and suggests further benefit when combined with corticosteroids.