All-Cause Mortality and Cardiovascular Outcomes With Prophylactic Steroid Therapy in Duchenne Muscular Dystrophy
All-Cause Mortality and Cardiovascular Outcomes With Prophylactic Steroid Therapy in Duchenne Muscular Dystrophy
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DOI：
10.1016/j.jacc.2012.12.008
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发表时间：
2013-03-05
影响因子：
24
通讯作者：
Khairy, Paul
中科院分区：
文献类型：
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作者：
Schram, Gernot;Fournier, Anne;Khairy, Paul
目的：本研究旨在确定类固醇治疗对杜氏肌营养不良（DMD）患者心肌病和死亡率的影响。DMD是一种使人衰弱的x连锁疾病，每3500名男孩中就有1人患有此病。虽然类固醇能减缓肌肉骨骼损伤，但对心脏功能和死亡率的影响尚不清楚。方法我们对肾素-血管紧张素-醛固酮系统拮抗剂联合或不联合类固醇治疗的DMD患者进行了一项队列研究。结果86例患者，年龄9.1±3.5岁，随访11.3±4.1年。接受类固醇治疗的63例患者中有7例（11%）死亡，而未接受类固醇治疗的23例患者中有10例（43%）死亡（p = 0.0010）。接受类固醇治疗的患者在随访5年、10年和15年时的总生存率分别为100%、98.0%和78.6%，而未接受类固醇治疗的患者的总生存率分别为100%、72.1%和27.9% （log-rank p = 0.0005）。在多变量倾向调整分析中，类固醇使用与死亡率降低76%相关（风险比：0.24；95%可信区间：0.07至0.91;p = 0.0351）。死亡率降低的原因是心力衰竭相关死亡减少（0%对22%，p = 0.0010）。在多变量分析中，类固醇与新发心肌病发生率降低62%相关（风险比：0.38；95%可信区间：0.16 ~ 0.90;p = 0.0270）。类固醇治疗患者左心室射血分数（-0.43% vs -1.09%, p = 0.0101）和缩短分数（-0.32% vs -0.65%, p = 0.0025）的年下降率较平缓。左室舒张末期尺寸的增加幅度较小（-0.47 vs -0.92 mm /年，p = 0.0105）。结论：在DMD患者中，类固醇治疗与全因死亡率、新发和进展性心肌病的显著降低相关。[J]中华医学会心脏科杂志，2013;31 (1):948- 954
Objectives This study sought to determine the impact of steroid therapy on cardiomyopathy and mortality in patients with Duchenne muscular dystrophy (DMD).Background DMD is a debilitating X-linked disease that afflicts as many as 1 in 3,500 boys. Although steroids slow musculoskeletal impairment, the effects on cardiac function and mortality remain unknown.Methods We conducted a cohort study on patients with DMD treated with renin-angiotensin-aldosterone system antagonists with or without steroid therapy.Results Eighty-six patients, 9.1 +/- 3.5 years of age, were followed for 11.3 +/- 4.1 years. Seven of 63 patients (11%) receiving steroid therapy died compared with 10 of 23 (43%) not receiving steroid therapy (p = 0.0010). Overall survival rates at 5, 10, and 15 years of follow-up were 100%, 98.0%, and 78.6%, respectively, for patients receiving steroid therapy versus 100%, 72.1%, and 27.9%, respectively, for patients not receiving steroid therapy (log-rank p = 0.0005). In multivariate propensity-adjusted analyses, steroid use was associated with a 76% lower mortality rate (hazard ratio: 0.24; 95% confidence interval: 0.07 to 0.91; p = 0.0351). The mortality reduction was driven by fewer heart failure-related deaths (0% vs. 22%, p = 0.0010). In multivariate analyses, steroids were associated with a 62% lower rate of new-onset cardiomyopathy (hazard ratio: 0.38; 95% confidence interval: 0.16 to 0.90; p = 0.0270). Annual rates of decline in left ventricular ejection fraction (-0.43% vs. -1.09%, p = 0.0101) and shortening fraction (-0.32% vs. -0.65%, p = 0.0025) were less steep in steroid-treated patients. Consistently, the increase in left ventricular end-diastolic dimension was of lesser magnitude (-0.47 vs. -0.92 mm per year, p = 0.0105).Conclusions In patients with DMD, steroid therapy is associated with a substantial reduction in all-cause mortality and new-onset and progressive cardiomyopathy. (J Am Coll Cardiol 2013;61:948-54) (C) 2013 by the American College of Cardiology Foundation