首页> 美国卫生研究院文献>BMJ Open >Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study
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Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study

机译:ACE抑制剂和β受体阻滞剂联合使用预防杜兴氏肌营养不良患儿心肌病的随机安慰剂对照试验? (DMD心脏保护研究):方案研究

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摘要

IntroductionAlthough cardiologists were ‘late-comers’ to the multidisciplinary team—contributing to the complex care of patients with Duchenne muscular dystrophy (DMD), they now recognise the importance of systematic cardiac surveillance and timely therapy to prolonged survival in patients with DMD. Empirical deployment of cardioactive medications has already improved outcomes, but the evidence base for clinical decision making is weak. Fundamental questions remain as to whether prophylactic therapy is justified and convincingly superior to prompt deployment of the same therapies once left ventricular (LV) dysfunction is detected. Even if it were, at what age should therapy be introduced and with what specific drugs?
机译:简介尽管心脏病专家是多学科团队的“新来者”,为杜兴氏肌营养不良症(DMD)患者的复杂护理做出了贡献,但他们现在认识到系统的心脏监测和及时治疗对于DMD患者延长生存期的重要性。对心脏活性药物的经验性部署已经改善了结局,但是临床决策的证据基础薄弱。一旦发现左心室功能不全,预防性疗法是否合理且具有说服力优于立即采用相同疗法的根本问题仍然存在。即使是,应在几岁时开始治疗,并使用哪种特殊药物?

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