Cardiac Management of the Patient With Duchenne Muscular Dystrophy
Cardiac Management of the Patient With Duchenne Muscular Dystrophy
复制标题
DOI：
10.1542/peds.2018-0333i
复制
发表时间：
2018-10-01
期刊：
影响因子：
8
通讯作者：
Olson, Aaron K.
中科院分区：
文献类型：
--
作者：
Buddhe, Sujatha;Cripe, Linda;Olson, Aaron K.
杜氏肌营养不良症 (DMD) 会导致进行性心肌病，导致显着的发病率和死亡率。为了提高 DMD 患者的生活质量，心脏护理的重点是监测和管理，目标是减缓心力衰竭并发症的发生和进展。本文旨在对用于为 2018 年 DMD 护理注意事项建议提供信息的心脏管理数据进行扩展回顾，并讨论临床争议和未来的管理方向。新的心脏指南包括有关心功能无创成像监测和药物治疗的变化。许多新兴疗法缺乏足够的循证数据，无法在 2018 年 DMD 护理注意事项中得到推荐。本文将这些作为临床争议和未来方向进行讨论。重要的新兴疗法包括新型心力衰竭药物、心室辅助装置的机械循环支持、心脏移植和心内除颤器。未来的研究应集中于这些先进疗法对 DMD 患者的风险和益处。我们通过简要讨论心脏与最近开发的药物之间的关系来结束本次综述，这些药物用于直接针对 DMD 中肌营养不良蛋白的缺失。
Duchenne muscular dystrophy (DMD) results in a progressive cardiomyopathy that produces significant morbidity and mortality. To improve the quality of life in patients with DMD, cardiac care is focused on surveillance and management, with the goal of slowing the onset and progression of heart failure complications. The current article is intended to be an expanded review on the cardiac management data used to inform the 2018 DMD Care Considerations recommendations as well as be a discussion on clinical controversies and future management directions. The new cardiac guidance includes changes regarding noninvasive imaging surveillance of cardiac function and pharmacologic therapy. Many emerging therapies lack sufficient evidence-based data to be recommended in the 2018 DMD Care Considerations. These are discussed in the present article as clinical controversies and future directions. Important emerging therapies include new heart failure medications, mechanical circulatory support with ventricular assist devices, heart transplantation, and internal cardiac defibrillators. Future research studies should be focused on the risks and benefits of these advanced therapies in patients with DMD. We conclude this review with a brief discussion on the relationship between the heart and the recently developed medications that are used to directly target the absence of dystrophin in DMD.